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USF Health has established a Neurocardiogenetics Clinic focused on improving the quality of life for patients with hereditary neuromuscular disorders who are at risk for or already experiencing cardiac complications. Innovative genetic research is a central component of the new clinic.

The multidisciplinary clinic teams faculty and staff with clinical expertise in cardiology, genetics and neurology. The clinic is directed by Aarti Patel, MD, assistant professor of cardiology and director of the Cardiac Imaging Fellowship Program at USF Health in collaboration with Thomas McDonald, MD, professor of cardiology and molecular pharmacology and physiology, and a member of the USF Health Heart Institute, and Theresa Zesiewicz, MD, professor of neurology and director of the USF Health Ataxia Research Center. Patients receive a complete cardiac examination and testing along with a comprehensive family genetic evaluation from providers familiar with their neurological history.

“Combining all that information, we are able to come up with a personalized treatment plan,” Dr. Patel said.

Many patients initially seen at the Neurocardiogenetics Clinic have been diagnosed with Friedreich’s ataxia (FA), a rare, debilitating and life-shortening neuromuscular disorder that usually strikes in childhood. At some point in their lives 55 to 60-percent of FA patients develop some form of cardiac disease. The most devastating of these is cardiomyopathy, a heart muscle disease that limits blood pumped to the rest of the body and increases risk for heart failure and abnormal heart rhythms.

“FA is a multisystem disease that affects many different parts of the body, including the heart,” Dr. Zesiewicz said. “And one of its most sinister complications is cardiomyopathy, an enlarged heart.”

The Neurocardiogenetic clinic offers patients and their families the opportunity to participate in ongoing genetic studies exploring potential links between heart disease and neuromuscular diseases like FA and certain types of other ataxias and muscular dystrophies.

Dr. Aarti Patel (left), assistant professor of cardiology, collaborates with Dr. Thomas McDonald, professor of molecular pharmacology and physiology, at the new USF Health Neurocardiogenetics Clinic. Dr. Theresa Zesiewicz. professor of neurology (not pictured), is another collaborator.

While a deficiency in the frataxin protein can lead to fibrosis and scarring of heart muscle tissue in FA, competing theories exist about how a genetic mutation may actually result in heart problems in this particular ataxia and other neuromuscular disorders.

Dr. McDonald’s research team is searching for answers to many questions, including how different genetic variations may lead to cardiac abnormalities and affect the severity of heart disease.

Small blood samples are collected from patient volunteers with different inherited neuromuscular and/or cardiac disorders, as well as family members who are unaffected genetic carriers of neuromuscular diseases. In laboratory cell culture dishes, USF Health researchers genetically reprogram the blood cells into pluripotent stem cells that can grow into any cell type. Then they induce these stem cells — containing the same genetic make-up as the patient who provided blood — to become nerve cells and heart muscle cells.

The goal is to work out at a molecular level how FA or other inherited neuromuscular diseases damage the heart muscle. Once that is achieved, Dr. McDonald said, the “disease in a dish” can be used to identify potential drug targets and test treatment options on the patient’s own cells.

“With stem cell technology and disease-in-a-dish modeling, we’re looking for the earliest changes in either a heart or a nerve cell (that lead to disease),” he said. “A big unanswered question is whether the molecular or cellular process happening in the nerve is the same as that damaging the heart muscle. By using stem cell-based research we hope to be able to determine if there is a connection between the nerve and the heart – or whether it’s a separate, independent process but based upon the same genetic defect.”

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The collaborative research builds upon robust data collected by doctors in the clinic, including the wide range of symptoms seen in patients with hereditary neurological disorders involving cardiac complications.

Ultimately, that can help improve understanding of why genetic variations in certain neuromuscular diseases cause cardiac complications, Dr. McDonald said. “Having a clear clinical picture of each research participant will help us correlate the genetics and function of cells we see in the laboratory with how that translates to disease progression in the individual.”

“With careful clinical surveillance and novel biomarkers, our hope is that we will be able to predict which patients and their family members are at highest risk for cardiovascular disease so we can intervene early with effective treatment,” Dr. Patel said. “We’re hoping to identify patients even before they develop cardiac symptoms, so we can prevent heart disease.”

-Video and photos by Allison Long, USF Health Communications and Marketing

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The Friedreich’s Ataxia Scientific Symposium: Understanding a Cure recently marked its 9th year of bringing dedicated scientists, clinicians, and industry partners with patients and their families to USF – all whom share a commitment to finding effective treatments and a cure for Friedreich’s ataxia and related disorders.  The Tampa Bay region is considered an epicenter for raising funds to support  research for Friedreich’s ataxia, or FA.  This year’s 10th Friedreich’s Ataxia Research Alliance (FARA) Energy Ball, held Saturday, Sept. 29,  following the symposium and the association’s international FA biomarkers meeting, raised a total of $2.5 million to help bridge the gap between discoveries and treatment.

Hosted by FARA and the USF Ataxia Research Center, the scientific symposium drew an audience of 700 — both live at the USF Health Center for Advanced Medical Learning and Simulation (CAMLS) and viewing the event in real-time through YouTube.

From left: Paul Avery, Dr. Theresa Zesiewicz, Dr. Charles J. Lockwood, Dr. Clifton Gooch, Jennifer Farmer, and Ron Bartek

FA is a rare inherited neuromuscular disease, typically diagnosed in children and adolescents, that progressively robs patients of their ability to walk, speak and function and can ultimately lead to early death. Currently, no drugs are approved for the treatment of FA.

Symposium participants discussed the latest FA translational research progress, as well as heard individual patient perspectives and from scientific and industry partners working with academia to move promising therapies into the clinical trial pipeline.  Researchers are attacking the devastating disease using multiple approaches, including working to improve mitochondrial function and inhibit inflammation, to replace the protein (frataxin) deficient in individuals with FA, and to combine gene correction with hematopoietic stem cell therapy. They also look for targeted biomarkers to help clinicians better assess disease progression and the response of individual patients to investigational FA drugs.

USF Health is one of 11 sites in Friedreich’s Ataxia Collaborative Clinical Research Network, an international network of clinical research centers working together to advance treatments and clinical care for those living with FA.  The USF Ataxia Research Center participates in the FA patient registry, the only worldwide registry containing demographic and clinical information on more than 2,000 patients with Friedreich’s ataxia.

Here’s a sampling of what some symposium speakers had to say:

Charles J. Lockwood, MD, senior vice president of USF Health and dean, USF Health Morsani College of Medicine

“Since 2008, this symposium continues to be a major annual event broadcast around the world… We will never stop looking for answers. We will never stop looking for a cure.”

Dr. Zesiewicz, center, with her USF Ataxia Research Center team

Theresa Zesiewicz, MD, professor of neurology and director, USF Ataxia Research Center

“At USF we have several active trials for FA, and we are close to finding a treatment to arrest disease progression and help alleviate some of the symptoms of FA. We are working towards offering even more clinical research in the future.”

From left, the Patient Panel, moderated by FARA Communications Director Felicia DeRosa, included: Laurel Avery of Florida, Sam Hill of Maryland, Annie Hamilton of New York, and Allison Dana of South Carolina.

Patient Panel:  Living with FA and Research Participation

Laurel Avery, 23, Tampa, FL, currently working on her master’s degree in entrepreneurship

“When I first started college, people automatically assumed that I need help with everything.  That’s a misconception, because the FA community is very adaptable and everyone works very hard to be resilient and strong.”

Sam Hill, 14, Annapolis, MD

“I’m proud that I’m able to keep up at school, even though I get tired easily.  I like math and science.”

Advocacy Organization Panel

Sanjay Bidichandani, PhD, FARA scientific director and head of genetics at the University of Oklahoma College of Medicine Department of Pediatrics

“From the beginning of when the FA gene was found until today, all the discoveries are now nodes or targets for therapies being developed.”

Advocacy Organization Panel participants included, from left: Sanjay Bidichandani, PhD, FARA scientific director; FARA President Ron Bartek; and Pat Ritschel, FARA board director

Ron Bartek, FARA president and co-founder

“We realized from the get-go that to develop a treatment and cure for this disease we’d need to build relationships with patients and their families, with academic investigators, with government partners at the NIH, FDA and Congress, and with industry partners.  With our encouragement, all of those stakeholders are now members of the family… Collaboration is FARA’s middle name.”

Pat Ritschel, FARA Board director

“By spending our effort to remove roadblocks, we attract more companies willing to work in this space of FA research… because the way forward is clear and they can see a path to the clinic and (drug) approval.”

The Scientific Panel included, sitting from left: Carole Ben-Maiman, MD, CEO of Chondrial Therapeutics; Stephanie Cherqui, PhD, associate professor at UC San Diego; Hao Wang, PhD, senior scientific director at Clinical Neuroscience-CNS TAU, Takeda Pharmaceuticals; and Kara Eichelkraut, senior manager of patient advocacy for Reata Pharmaceuticals.

Scientific Panel – FA Treatment Approaches

Jennifer Farmer, FARA executive director

“We have lots of different treatment approaches in various stages of development.  This is very important, because we know that there is no disease fully treated with just one drug or one therapy.”

Hao Wang, PhD, senior scientific director of Clinical Neuroscience-CNS TAU, Takeda Pharmaceuticals

“The patients have educated us about their diagnosis and journey, and what kind of treatments they need. They have a great sense of humor despite the difficulties they are experiencing… The feedback we received from them has helped with study design.”

(Takeda’s investigational drug candidate TAK-831 is in a phase 2 clinical study to evaluate the enzyme inhibitor’s effectiveness on upper extremity motor function and manual dexterity.)

Kara Eichelkraut, senior manager of patient advocacy, Reata Pharmaceuticals

“Anything and everything anyone in this room does to contribute to FA research makes a difference.  If it’s participation in a clinical trial that’s great, but if you cannot do that there are other things you can do, like providing a cheek swab or giving a vial of blood.”

(Reata received orphan drug designation for omaveloxolone for investigational treatment of FA by the FDA and EMA. FARA is enrolling 100 patients in a phase 2, part 2 clinical trial.)

Closing Comments

Clifton Gooch, MD, chair of neurology, USF Health Morsani College of Medicine, thanked participants for their courage and inspiration

“Twenty years after the founding of FARA we have over a dozen clinical trials focused on getting at the very heart of this disease in the genes themselves, using techniques of genetic engineering and modulation, therapies to support the mitochondria, and others… There is great reason for hope.

“Once we find the genetic therapy that treats this disease we will have the tools to prevent it in those who have the aberrant gene, to fix the gene so that they never develop the disease… That’s the world we’re working towards. We want to make FA a footnote in the medical literature, like smallpox.”

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-Videos by Torie M. Doll, and photos by Eric Younghans, USF Health Communications and Marketing

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Patients have always been the center of the yearly scientific symposium hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the University of South Florida Ataxia Research Center.

But, for the 8^th annual symposium held Sept. 15 at USF’s Gibbons Alumni Center, patients took on an even more prominent role. The panel discussion in which they share their stories about living with the rare, but devastating, progressive neurodegenerative disease, including patient participation in clinical trials, was moved up in the program format.

The 8th annual scientific symposium hosted by FARA and USF was held in the USF Gibbons Alumni Center.  More than 750 in the FA community listened to the latest perspectives on ataxia research from patients and scientists, in person and internationally via live-stream.

And this year, led by FARA spokesperson Kyle Bryant as moderator, the four patient panelists were the ones driving the conversation with leading researchers from academia and industry who sat onstage beside these young adults to discuss the latest advances in the search for effective treatments and, ultimately, a cure.

More than 250 attendees gathered at the USF Gibbons Alumni Center for the symposium, which was also live-streamed and viewed worldwide by those in the FA community, over 500 people in eight countries. The symposium “Understanding Energy for a Cure” kicked off a series of events in Tampa Bay to raise awareness about FA, culminating Sept. 17 with the FARA Energy Ball gala, which this year raised $2 million to benefit innovative ataxia research.

The patient panel, moderated by FARA spokesperson Kyle Bryant (far left), helped drive the conversation with leading researchers. Participants were, from left, Alex Fielding, Sean Baumstark, Alison Avery and Anna Gordon.

“My parents and sister never really let me believe that Friedreich’s ataxia was going to stop me,” said panelist Alison Avery, 22, diagnosed with Friedreich’s ataxia at age 18, who is interning with the National Football League’s social responsibility department in NYC following college graduation. “It may have changed the way that I do certain things, but right now I’m living on my own in New York City, and that’s something not everyone would do, whether or not they have FA.”

Alison participates in the “Cardiac MRI and Biomarkers in Friedreich’s Ataxia” study at Children’s Hospital of Philadelphia and another evaluating the relationship between exercise performance and neurological/cardiac status and overall functioning in children and adults with FA. “I’m excited to be able to share my perspective on being involved in different research studies,” she said. “I feel like that’s something more people should know, especially the researchers — about how patients actually feel about trials and studies.”

USF System President Judy Genshaft said USF has made neurosciences, including ataxia research, a high institutional research priority.

Friedreich’s ataxia typically strikes in childhood or adolescence and leads to a progressive loss of coordination and muscle strength, eventually robbing young people of their energy and ability to walk. While the neurological symptoms are most visible, FA is a multisystem disease that can adversely affect cardiac function, metabolism, vision, hearing and the skeletal system. There is currently no approved treatment for FA.

“Throughout the history of this event, the one constant has been how incredibly motivating and inspiring it is to hear from patients and their families who never fail to share one valuable message: ‘Live life to its fullest despite the challenges of Friedreich’s ataxia,’” said USF System President Judy Genshaft in her symposium welcome remarks.

FARA Executive Director Jennifer Farmer introduced the patients and provided insights on their participation in studies and clinical trials.

The USF Health Morsani College of Medicine is one of 10 sites in the international FARA Collaborative Clinical Research Network, all working to discover treatments that can attack FA on different fronts and improve the quality of life for patients.

“We’ve made this a high research priority within the institution,” President Genshaft said. “Over the last 20 years FARA’s international collaborative of researchers has increased the pace in the fight against FA. Today more than 20 drugs are in the treatment pipeline and ongoing studies are working toward the discovery of new therapies… We have every reason to be hopeful, but we do know there is more work to be done.”

Theresa Zesiewicz, MD, professor of neurology and director of the USF Ataxia Research Center, presented promising preliminary results from two clinical trials conducted at USF, among other sites. – Photo by Kent Ross

Theresa Zesiewicz, MD, professor of neurology and director of the USF Ataxia Research Center, updated attendees on the center’s initiatives.

“We started out at USF with one clinical trial eight years ago, and now we have five or six clinical trials and each (investigational) drug works differently,” Dr. Zesiewicz said. “Some drugs work to increase frataxin (the protein depleted in those with FA), some drugs work on inflammation, some work as strong antioxidants. So, there may not be one magic bullet to stop this disease; rather, it may require a cocktail of therapies, a conglomerate of different compounds to help delay or stop the disease process.”

David Lynch, MD, PhD, (center) lead investigator for the FA Natural History Study at Children’s Hospital of Philadelphia, responds to a patient question. He was joined in the discussion of clinical trials by Martin Delatycki, PhD, (far left) of Murdoch Children’s Research Institute, Melbourne, Australia.

Some promising preliminary results for two clinical trials conducted at USF, among other sites, were announced by lead investigator Dr. Zesiewicz. Both studies were done in collaboration with FARA.

• EPI-743 Safety and Effectiveness Study: The Phase 2 open-label extension study, sponsored by Edison Pharmaceuticals, tested the effectiveness of the potent antioxidant EPI 743 primarily on vision, and secondarily, on neurological function in adult patients with FA. After two years of study and a year of data analysis, the researchers found that patients taking EPI-743 from the study’s start demonstrated markedly less disease progression than would be expected in the natural history of the disease. The improvement in neurological function was dose-dependent, and although the last 18 months of the study were open-label, patients and investigators were blinded to the drug dose allocation. Additional studies of EPI-743 are planned in pediatric patients and those with point mutations.

FARA President Ron Bartek thanked everyone in the room, including researchers, pharmaceutical partners and patients and their families, for working together to advance discoveries to “slow, stop and reverse” Friedreich’s ataxia.

• Retrotope RT001 Phase 1/2: The randomized double-blind, placebo-controlled trial evaluated the safety, tolerability and early effectiveness of the stabilized fatty acid RT001 in adult patients with FA. In the small, 28-day study, researchers found that the drug was safe, well tolerated at high doses and rapidly absorbed to target levels, with early signs of effectiveness. Earlier this year, the FDA granted Retrotope orphan drug designation for RT001 in FA.

The scientist and physician panelists at the symposium covered four areas of FA research:

• Basic and Discovery Science: Helene Puccio, PhD; Marek Napierala, PhD; and Jordi Magrane, PhD
• Drug Development and Advancing Treatments: Mark Payne, MD; and Barry Byrne, MD, PhD.
• FA Biomarkers: Kimberly Lee Lin, MD; Angel Martin, PhD student; and Christophe Lenglet, PhD.
• Clinical Trials and Translating Treatments to Improved Care: Martin Delatycki, PhD; and David Lynch, MD, PhD.

The researchers discussed their scholarly work, progress beyond their laboratories and its relevance to advancing treatments. They also emphasized their passion for FA science and personal commitment to patients.

Scientists participating in the Basic and Discovery Science panel discussion were, from left, Jordi Magrane, PhD, of the Brain and Mind Research Institute, Weill Cornell Medical College; Marek Napierala, PhD, of the University of Alabama; and Helene Puccio, PhD, of the Institute of Genetics and Molecular and Cellular Biology, University of Strasbourg.

Moving from treating symptoms to slowing and stopping progression to reversing disease is “not an overnight event,” said David Lynch, MD, PhD, lead investigator for the FARA Natural History Study at Children’s Hospital of Philadelphia. “So, in 15 years we may look back and talk not about the advance but about the 15 advances from each of 15 clinical trials superimposed on top of one another, eventually leading to that four letter word — cure.”

Answering patient questions on drug development and advancing treatments were physician-scientists Barry Byrne, MD, PhD, (left) of the University of Florida College of Medicine; and R. Mark Payne, MD, of Indiana University School of Medicine.

Despite the challenges, the researchers agreed that the steadfast determination and resilience of patients and their families energizes them to keep working toward a cure.

“Everything we do is for the patients, and we are all in this together trying to find a treatment and cure for Friedreich’s ataxia,” said USF’s Dr. Zesiewicz. “That’s the only reason we’re here.”

Participants in the FA BioMarkers panel discussion were, from left, Kimberly Lee Lin, MD, of Children’s Hospital of Philadelphia; Christophe Lenglet, PhD, of the Institute for Translational Neuroscience, University of Minnesota; and Angel Martin, a PhD candidate at Duke University.

Alison Avery, second from right, credits her family — sister Laurel Avery (left) and parents Paul and Suzanne Avery — with “never really letting me believe that Friedreich’s ataxia is going to stop me.”

Dr. Zesiewicz with members of the USF Ataxia Research Center, one of 10 sites in the international FARA Collaborative Clinical Research Network. – Photo by Kent Ross

Photos by Eric Younghans and video by Sandra C. Roa, USF Health Communications and Marketing

International experts from academia and industry will gather to discuss advances in Friedreich’s ataxia from the laboratory to the clinic.

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Tampa, FL (Sept. 12, 2016) — The University of South Florida (USF) will bring together leading researchers and patients searching for a treatment for Friedreich’s ataxia (FA) and related disorders at the eighth annual scientific symposium “Understanding Energy for A Cure.” The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 15, at the USF Sam and Martha Gibbons Alumni Center, 11810 USF Alumni Drive, Tampa, FL 33620.

The event, free and open to the public, is hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the USF Ataxia Research Center.

The symposium will include four scientific panel discussions moderated by FARA spokesperson Kyle Bryant. The topics will be:

• Basic and Discovery Science
• Drug Development and Advancing Treatments
• FA Biomarkers
• Clinical Trials and Translating Treatments to Improved Care

Before the scientific panel discussions, Dr. Theresa Zesiewicz, professor of neurology and director of the USF Ataxia Research Center, will update attendees on the Friedreich’s ataxia initiatives at the center. USF is one of 10 sites in the international FARA Collaborative Clinical Research Network.

Patients will share their experiences about living with FA in a discussion moderated by Jen Farmer, executive director of FARA.

Each year, the Friedreich’s Ataxia Scientific Symposium draws patients and their families to USF from across the country to share their thoughts and perspectives about energizing the search for a cure.  The event will also be attended by supporters of the FARA Energy Ball gala, held on Saturday, Sept. 17.

For the first time this year, on Friday Sept. 16, USF will also host FARA’s 4^th Biomarkers Meeting, which attracts international scientists to focus on measurable metabolic, neurological and cardiac indicators of Friedreich’s ataxia as well as endpoints for the disease.

Thursday’s “Understanding Energy For A Cure” symposium will be broadcast through the Ustream’s CureFA channel, with opportunities for visitors to join the conversation long distance. To watch the presentations in real-time, visit http://www.ustream.tv/channel/curefa on Sept. 15 at 6 p.m. EST. A Ustream account/membership is not needed to join.

Friedreich’s ataxia is a rare, progressive neurodegenerative disease affecting children and adults for which there is currently no approved therapy. Symptoms include neurologic, cardiac, orthopedic, and endocrine dysfunction.

For more information or to RSVP, please visit www.curefa.org/events/event/337-understanding-energy-for-a-cure-symposium, or call (813) 974-5909.
                                                                                           – About USF Health –

USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Physical Therapy and Rehabilitation Sciences, the Biomedical Sciences Graduate and Postdoctoral Programs, and the USF Physicians Group. The University of South Florida is a Top 50 research university in total research expenditures among both public and private institutions nationwide, according to the National Science Foundation. For more information, visit www.health.usf.edu

                                                – About The Friedreich’s Ataxia Research Alliance (FARA) –

FARA is a non-profit organization dedicated to curing FA through research. FARA grants and activities provide support for basic and translational FA research, pharmaceutical/biotech drug development, clinical trials, and scientific conferences. For more information, go to www.curefa.org.

Media contact:
Anne DeLotto Baier, USF Health Communications
abaier@health.usf.edu or (813) 974-3303

Hosted by FARA and the USF Ataxia Research Center, the annual scientific symposium emphasizes a patient-centered approach to research

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For the first time in its seven-year history, the Friedreich’s Ataxia Scientific Symposium brought together several pharmaceutical industry leaders to discuss preclinical and clinical studies.  The companies are all attacking Friedreich’s ataxia (FA) on different fronts with the same goal in mind:  to get the first treatment for the rare, but devastating, neuromuscular disease approved and on the market as soon as possible.

The Sept. 17 symposium, hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the USF Ataxia Research Center, drew an audience totaling more than 500, both live at the USF Marshall Center Ballroom and viewing the event in real-time through Ustream’s CureFA channel.

The symposium featured a discussion by leading representatives of pharmaceutical and biotechnology companies working with FARA and academia to conduct new research attacking Friedreich’s ataxia on several fronts.

Representatives and supporters of FARA, the research community, and patient and their families – many whom attended FARA’s Energy Ball gala on Saturday evening, Sept. 19 — were welcomed by USF President Judy Genshaft.

The translational center of excellence at USF is “one of the most active in the world, testing potential new drugs for Friedreich’s ataxia,” President Genshaft said. “We are unstoppable in the fight for a cure for FA.”

In the last seven years, the staff of the USF Ataxia Research Center, focused on identifying and developing effective treatments for inherited ataxia disorders, has expanded to eight, including two clinicians, a fellow, nurses and research coordinators.  Center Director Theresa Zesiewicz, MD, professor of neurology, gave an overview of the center’s eight clinical trials — six active and two finishing up. USF, one of 10 sites in the international FARA Collaborative Clinical Research Network, is recruiting patients for two of the three trials presented by pharmaceutical industry leaders at the symposium. The USF center is also working with Agilis, one of the biopharmaceutical companies at the symposium, to develop their gene therapy protocol, expected to be submitted in 2016 to the Food and Drug Administration (FDA).

USF President Judy Genshaft welcomed attendees to the USF Tampa campus for the seventh annual scientific symposium “Understanding Energy for A Cure,” hosted by the Friedreich’s Ataxia Research Alliance and the USF Ataxia Research Center.

Friedreich’s ataxia is triggered by a single genetic defect that limits production of frataxin, a protein vital to the function of the energy-producing factories, or mitochondria, of the cell. This leads to a variety of symptoms including neurodegeneration that can cause muscle weakness and loss of coordination and balance, energy deprivation and fatigue, vision impairment, slurred speech, aggressive scoliosis, diabetes and life-shortening cardiac disease. Most young people diagnosed with Friedreich’s ataxia require a cane, walker or wheelchair by their teens or early 20s. There are currently no approved treatments.

FARA President Ron Bartek and Executive Director Jennifer Farmer spoke about the progress in Friedreich’s ataxia research worldwide and the value of the organization’s 2,600-member patient registry in bringing together all its stakeholders.

FARA’s Executive Director Jennifer Farmer and President Ron Bartek.

Featured speaker Sanjay Bidichandani, MBBS, PhD, the chair of pediatric medical genetics at the University of Oklahoma College of Medicine and member of the FARA Board of Directors, was part of the team that first identified the Friedreich’s ataxia gene in 1996.  Fueled by resources and partnerships cultivated by FARA, Dr. Bidichandani said, the understanding of the disease process advanced relatively quickly since that genetic discovery and has yielded robust expansion of investigational drugs in the treatment pipeline.

A panel moderated by Dr. Bidichandani featured representatives from four biotechnology and pharmaceutical companies conducting Friedreich’s ataxia research in collaboration with FARA and academia – Jeffrey Sherman, MD, executive vice president for research and development and chief medical officer, Horizon Pharma; Jodi Cook, PhD, vice president of operations, Agilis Biotherapeutics; Colin Myer, MD, chief medical officer, Reata Pharmaceuticals; and Robert De Jager, MD, chief medical officer, Retrotope. Their newly activated studies cover a range of therapeutic targets, including finding ways to boost frataxin production, improving mitochondrial function, reducing mitochondrial damage and oxidative stress, and delivering gene therapy.

Featured speaker Dr. Sanjay Bidichandani, a member of FARA’s Board of Directors, was part of the group that discovered the gene for Friedreich’s ataxia in 1996. He gave an overview of the tremendous progress in less than 20 years leading to a robust treatment pipeline.

“FARA has really galvanized the patient community, academia, industry and even regulators to develop better insights into this disease and how we can all work together,” said Horizon Pharma’s Dr. Sherman. “At the end of the day, they’ve really brought to the forefront the importance of patient centricity and the voice of the patient in clinical and basic science research.”

Horizon Pharma has repurposed a drug already approved by the U.S. Food and Drug Administration for use in treating two other rare genetic disorders, chronic granulomatous disease and severe malignant osteopetrosis. The company recently launched a Phase 3 randomized, double-blind, placebo-controlled trial to test the safety and effectiveness of Actimmune® (interferon gamma-1b) in improving neurological function in 90 Friedreich’s patients at four U.S. sites. Previous research indicated that Actimmune®, which mimics a protein made by the body to help prevent infection, increases frataxin levels to reduce nerve cell depletion and muscle atrophy.

The symposium brought together representatives and supporters of FARA and the research community with patients in their families.

Agilis focuses on engineering and delivering therapeutic DNA to replace the damaged frataxin gene. Preclinical studies are employing a safe virus to optimally deliver the corrective gene to key targets, allowing safe and effective long-term expression of the frataxin protein.

Earlier this year Reata began a Phase 2 randomized, placebo-controlled, double-blind trial testing the safety and effectiveness of various dose levels of the oral medication RTA 408 in treating Friedreich’s ataxia. Known as the MOXle study, the trial is enrolling patients at sites worldwide, including USF. Preclinical studies have shown that RTA 408 directly activates antioxidative pathways to improve mitochondrial function.

This month USF enrolled the first of 18 patients in Retrotope’s 28-day randomized, double-blind controlled trial evaluating the safety of the investigational oral drug RT001 in ambulatory patients with Friedreich’s ataxia. USF and the University of California Los Angeles will be the only two sites for the Phase 1 study. The compound RT001 is a stabilized fatty acid shown to shut down the toxic free radical degradation of polyunsaturated fats, an essential component of cell membranes, and reduces further damage to the mitochondria.

Jade Perry, a member of the patient panel, was accompanied by her service dog Bo, a labradoodle.

The scientific panel was followed by a question-and-answer session on patients’ perspectives of living with Friedreich’s ataxia.  The panel was moderated by symposium host Clifton Gooch, MD, professor and chair of the Department of Neurology at the USF Health Morsani College of Medicine.

The four patient participants, all diagnosed in their teens or early 20s, emphasized a common theme – that they choose every day to carry on and live life to its fullest  despite the challenges of Friedreich’s ataxia.

Jade Perry, 25, was accompanied to the stage by the service dog she trained, a labradoodle named Bo.   Perry, who is finishing up a master’s degree in education from Coastal Carolina University, recently got her first full-time teaching job.

“I love riding my trike and try to keep my schedule packed so FA can’t slow me down,” she said.

Kendall Harvey, center, was diagnosed with later-stage Friedreich’s ataxia three years ago at age 25. “In spite of my diagnosis, my family and I are living life to the fullest,” she said.

Kendall Harvey, 28, diagnosed at age 25 with later-onset Friedreich’s ataxia, still walks unassisted.  She and her husband live in Austin, TX, with their 11-month old son Brooks.

Harvey, who participated in volleyball, track and other sports as a youth, said she noticed problems with agility and balance while taking dance lessons in preparation for her 2013 wedding.  She chalked it up to getting a little older or “being out of shape.”  But continuing symptoms finally led her to a neurologist who ran a battery of tests, culminating with a full genetic panel.

“The first time I heard of FA was the day I was diagnosed,” Harvey said. “FA has changed my perspective. Now, I live more in the moment than worrying about all the things in the future.

“On the days when I stumble a little more, my speech is slurred and I’m angry that my body is not behaving the way I’d like, my son is a fantastic reminder that I’m still capable of amazing things.  He’s very humbling.”

In his closing remarks Dr. Clifton Gooch, professor and chair of neurology at the USF Health Morsani College of Medicine, encouraged continued collaboration in the fight to find effective treatments and a cure for Friedreich’s ataxia.

USF’s Dr. Gooch closed the symposium by emphasizing the tremendous progress made in the research and development of lead drug candidates for Friedreich’s ataxia and encouraging all to continue to carry on the fight against the disease with laser focus.

“When pharma becomes engaged, that means the research is good enough to put smart money behind the disease to get a drug to market,” Dr. Gooch said. “There is more than just a glimmer of hope. We’re on the cusp of great possibilities… We look forward to the day when Friedreich’s ataxia will become a historical footnote like smallpox.”

For more information on the FARA patient registry, which provides notices about new clinical trials, go to http://www.curefa.org/patient-registry

Dr. Gooch, second from left, moderated the question and answer session with patients, l to r, Jade Perry, Erin O’Neil, Kendall Harvey, and Chris Nercesian.

FARA ambassador Kyle Bryant with Dr. Theresa Zesiewicz, director of the USF Ataxia Research Center.

Kyle Bryant with Sam Bridgman, who recently received a full scholarship to attend the graduate program at the USF Muma College of Business.

L to R: FARA President Ron Bartek; Dr. Clifton Gooch, chair of USF Health Neurology; Dr. Jodi Cook, vice president of operations, Agilis Biotherapeutics; Dr. Robert Molinari, founder and CEO of Retrotope; Dr. Jeffrey Sherman, CMO, Horizon Pharma; Dr. Theresa Zesiewicz, director, USF Ataxia Research Center; and Dr. Colin Myer, CMO, Reata Pharmaceutical.

Photos by Eric Younghans, video by Sandra C. Roa, USF Health Communications and Marketing

Pharma, biotech leaders will discuss several new clinical studies testing drugs and gene therapy for Friedreich’s ataxia

//www.youtube.com/watch?v=EIMrtGI8Kfo

Tampa, FL (Aug. 31, 2015) — The University of South Florida (USF) will again bring together leading researchers and patients searching for a treatment for Friedreich’s ataxia and related disorders at the seventh annual scientific symposium “Understanding Energy for A Cure.”  The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 17, at the USF Marshall Student Center Ballroom, USF Cedar Circle, Tampa, FL  33620.

The event, free and open to the public, is hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the USF Ataxia Research Center.

For the first time, the symposium will include a panel discussion with several biotechnology and pharmaceutical industry leaders about new clinical trials testing drugs and gene therapy for Friedreich’s ataxia. The panelists include representatives from Agilis Biotherapeutics, LLC; Horizon Pharma, plc; Reata Pharmaceuticals Inc; and Retrotope, Inc. The companies collaborate with FARA and academic institutions to focus on research that will improve the quality and length of life for those diagnosed with Friedreich’s ataxia and lead to treatments that eliminate symptoms.

Patients with Friedreich’s ataxia and their families come to USF from across the country to share their thoughts and perspectives about energizing the search for a cure.  The event will also be attended by supporters of the FARA Energy Ball gala, held on Saturday, Sept. 19.

The scientific symposium will again be broadcast through Ustream’s CureFA channel, with opportunities for visitors to join the conversation long distance. To watch the presentations in real-time, visit http://www.ustream.tv/channel/curefa on Sept. 17 at 6 p.m. EST.  A Ustream account/membership is not needed to join.

Friedreich’s ataxia is a rare, progressive neurodegenerative disease affecting children and adults for which there is currently no approved therapy. Symptoms include neurologic, cardiac, orthopedic, and endocrine dysfunction.

The symposium will be hosted by Clifton Gooch, MD, professor and chair of the Department of Neurology in the USF Health Morsani College of Medicine. Theresa Zesiewicz, MD, professor of neurology and director of the USF Ataxia Research Center, will update attendees on the Friedreich’s ataxia initiatives at USF, one of 10 sites in the international FARA Collaborative Clinical Research Network.

FARA President Ron Bartek and Jennifer Farmer, FARA Executive Director, will give an overview of progress nationwide in Friedreich’s ataxia research.

Featured speaker Sanjay Bidichandani, MBBS, PhD, chair of pediatric medical genetics and professor of pediatrics and biochemistry and molecular biology at the University of Oklahoma College of Medicine and member of the FARA Board of Directors, will talk about the pipeline of investigational treatments for Friedreich’s ataxia.  He was part of the group that discovered the gene for Friedreich’s ataxia and, over the last 15 years, his research has helped characterize the disease’s genetic and epigenetic defect.

USF Health’s Dr. Gooch will moderate a question-and-answer session on patients’ perspectives of living with Friedreich’s ataxia and clinical trial participation.

For more information, please visit http://www.curefa.org/energyball, or call (813) 974-5909.

– About USF Health –

USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Physical Therapy and Rehabilitation Sciences, and the USF Physicians Group. USF Health is an integral part of the University of South Florida, a high-impact, global research university dedicated to student success. For more information, visit www.health.usf.edu

– About The Friedreich’s Ataxia Research Alliance (FARA) –

FARA is a non-profit organization dedicated to curing FA through research. FARA grants and activities provide support for basic and translational FA research, pharmaceutical/biotech drug development, clinical trials, and scientific conferences. For more information, go to www.curefa.org.

Media contact:
Anne DeLotto Baier, USF Health Communications
abaier@health.usf.edu or (813) 974-3303

Video by Sandra Roa, USF Health Communications

The fundraiser teams patient with USF neurologist who studies new treatments for the neurodegenerative disease

Tampa, FL (Feb. 18, 2015) – The first time his right arm froze up, Rick Karczewski let it pass.

Then six months later on Memorial Day, driving his Harley-Davidson motorcycle up Interstate 75 to meet fellow military veterans heading to Florida National Cemetery in Bushnell, his hands began to cramp. “There’s something wrong with me,” he thought. Karczewski told his buddies he’d better head back home.

His doctor referred him to Dr. Theresa Zesiewicz, a neurologist at the University of South Florida’s Parkinson’s Disease and Ataxia Center. A battery of exams eventually confirmed his suspicions: he had Parkinson’s disease.

USF neurologist Dr. Theresa Zesiewicz (on motorcycle) will participate in the motorcyle ride benefitting the USF Parkinson’s disease center named in honor of her mother, who died from complications of the neurodegenerative disease.  One of Dr. Zesiewicz’s patients helped organize the fundraiser.

Now 18 months later, Karczewski, 49, hopes to give something back to the doctor who sat with him during those first long visits talking through all of his questions, and who has continued to treat him ever since.

On Saturday, Feb.21, the inaugural Shakes, Rattles, Rides and Rods fundraiser will be held in Riverview, aiming to raise at least $10,000 for the Frances J. Zesiewicz Center for Parkinson’s Disease at USF, as well as the Michael J. Fox Foundation.

The Frances J. Zesiewicz Center was named in honor of Dr. Zesiewicz’s mother, who died from complications from the disease.

For Karczewski, the fundraiser and ride is a way for him to combine his passion for motorcycles with his desire to contribute to the center and respond to the disease in a positive manner that can help others.

“I’d rather be proactive instead of being down on myself that I have it,” Karczewski said. He added that 100 percent of the proceeds will go to the two foundations, with most of that geared for the USF center to give back to Dr. Zesiewicz for everything she’s done for him. He recalled that for his first office visit with her, she spent close to 45 minutes talking with him.

“When she’s with a patient, that patient gets her time, no matter how long it is,” he said.

Dr. Zesiewicz empathizes with the pain and frustration of patients. She had been a USF fellow in Movement Disorders in the USF Department of Neurology for about a year in 1994 when she was out at a restaurant with her mother. As they were dining, she saw her mother’s hands shaking. “What’s that?” Dr. Zesiewicz asked her. She went on to diagnose her mother with Parkinson’s disease. Frances Zesiewicz died 16 years later in 2010, followed by her brother – Dr. Zesiewicz’s uncle – nine weeks later from the same disease.

“I have a personal stake in this disease,” Dr. Zesiewicz said.

Parkinson’s disease is a neurodegenerative brain disorder that typically progresses slowly from slight tremors to impaired motor dexterity and life-changing problems, including alteration in balance and non-motor secondary symptoms such as depression and anxiety. At least one million people in the United States live with Parkinson’s disease. There is no known cure, though treatments such as medication and surgery are used to manage symptoms.

Dr. Zesiewicz is a tenured USF professor of neurology, as well as director of the USF Ataxia Research Center, the Frances J. Zesiewicz Center and Foundation for Parkinson’s Disease at USF, and the James A. Haley Veterans’ Administration Parkinson’s Disease Clinic.

Her research team has focused on motor and non-motor symptoms of neurological diseases and the testing of new medications to treat them, including currently a national clinical trial testing a powerful antioxidant medication in patients with Friedreich’s ataxia, a rare debilitating neuromuscular disease. This medication is also being tested in Parkinson’s disease.

Dr. Zesiewicz hopes to use donations from the upcoming fundraiser for physical therapy and tai chi classes to help Parkinson’s disease patients develop core strength to counteract problems with balance that can set in as the disorder advances.

When she heard about Karczewski’s plans for the fundraiser and that he and his fellow organizers, wife and support team planned to steer donations toward the center named for her mother, she felt extremely grateful.

“They have just been fantastic in raising money and awareness for this disease,” she said. “They decided to do it and never looked back. They are giving and loving people.”

Karczewski organized the fundraising event with Mike Savidge, publisher of Go For A Ride Magazine, after discovering that Savidge’s wife, Charlette, also has the disease. The two combined forces to aid efforts to develop further treatments and a cure for the disease.

Karczewski, who started riding minibikes when he was 8 years old, said he realizes he probably only has another year left to enjoy his passion. He already has downsized from a Harley-Davidson Electra Glide to a Softail. He can ride only about 40 to 50 miles at a time before his body gets too fatigued and he has to stop.

But he always plans to play a part in the fundraisers, which he says will become annual events.

For the first one, he expects to put Dr. Zesiewicz on the back of his bike.   “I thought it would be kind of cool to have her riding with me,” he said.

EVENT INFORMATION:
The inaugural Shakes, Rattles, Rides and Rods fundraiser to benefit foundations working toward treatments and a cure for Parkinson’s disease will be held from noon to 6 p.m. on Saturday, Feb. 21, at the Hidden River Travel Resort, at 12500 McMullen Loop in Riverview.

The event, hosted by AMVETS Post 44, American Legion Riders Alafia Post 148, and Go For A Ride Magazine, will first kick off with a motorcycle ride. Riders should register at the American Legion Alafia Post 148 at 7240 U.S. 301 in Riverview between 10 a.m. and 11:45 a.m.

Requested donations to the event are $15 per person or $25 per couple. It includes food, entertainment, music, dancing, a live auction, vendors, and a grand-prize drawing.

All proceeds go to the Frances J. Zesiewicz Center for Parkinson’s Disease at USF, as well as the Michael J. Fox Foundation.

For more information, call Rick Karczewski at (813) 810-5001.

News release by Saundra Amrhein

Media contact: Anne DeLotto Baier, USF Health Communications
abaier@health.usf.edu or (813) 974-3303

Tampa, FL (Dec. 2, 2014) — The University of South Florida (USF) Morsani College of Medicine is conducting a clinical research study on erectile dysfunction (ED) in patients suffering from Parkinson’s disease.  The principal investigator for the study is Dr. Theresa Zesiewicz, a medical doctor, professor of neurology and the director of clinical research for the USF Frances Zesiewicz Parkinson’s Research Foundation.

The new pilot study will test the safety and effectiveness of an investigational combination medication for ED in patients with Parkinson’s disease who fail to respond to current standard ED medications.  Enrollment begins this month.

While Parkinson’s disease is a movement disorder, the non-movement symptoms, including ED can present challenges to the normal activities of life.  ED is more common in men with Parkinson’s disease, with a reported prevalence rate of approximately 60 percent.  The current standard of care is oral treatment with phosphodiesterase-5 inhibitor (PDE-5i) medications, such as Cialis^®, Viagra^®, or Levitra^®; however, a large percentage of these patients fail to respond adequately to this treatment.

Dr. Theresa Zesiewicz

“Clearly, this is a patient population in need of improved treatments,” said Dr. Zesiewicz. “The non-motor symptoms of Parkinson’s disease are often more bothersome and hinder quality of life to a greater degree than the motor symptoms of the disease, including tremor and rigidity. It’s important that we continue to identify non-motor symptoms in our patients and develop new strategies to treat them.”

In the current clinical study, Dr. Zesiewicz will treat volunteer, Parkinson’s disease patients who have ED with a combination medication designed to help those who respond poorly to PDE-5i medications, such as Cialis^®, Viagra^®, or Levitra^®_.  All qualifying study participants will receive the active medications. Participant progress and the effectiveness of the medication will be monitored and assessed in comparison to baseline values. Sagene Pharmaceuticals Inc., of Oldsmar, FL, which holds the intellectual property for the combination medication, is helping to fund this study. The pilot study is designed to help determine the value of this product as a potentially new treatment for ED in the Parkinson’s population.

The USF Parkinson’s Disease Foundation and Clinical Research Center treats approximately 2,500 patients per year from the Tampa Bay region and surrounding areas. Located in the Carol & Frank Morsani Center for Advanced Healthcare on the USF Tampa campus, the center focuses on clinical care and new therapies for motor and non-motor symptoms of Parkinson’s disease. Dr. Zesiewicz and nurse practitioners Tracy Jones, ARNP, and Terry McClain, ARNP, provide state-of-the-art care to patients with Parkinson’s disease.

For more information, please call 813-974-5909 and ask to speak to Tanya Aranca or Kevin Allison.

About Sagene Pharmaceuticals

Sagene Pharmaceuticals is a biopharmaceutical company developing novel applications for combinations of FDA-approved drugs to treat ED. Sagene has shown that its patented combinations can enhance efficacy of current ED treatments by targeting multiple disease pathways not typically addressed by approved drugs. The combination approach for treating diseases with FDA-approved drugs to improve their therapeutic effect, reduces development risk, time and costs by utilizing the 505(b)(2) NDA approval process. Sagene has assembled a team of experts experienced in drug development, clinical trials and regulatory affairs. Sagene has conducted preclinical studies showing proof of concept and the benefits of combination therapy in laboratory animals and human platelets. Sagene is seeking funding partners to develop its ED combination in areas of unmet need such as Parkinson’s disease and diabetes. The company is headquartered in Oldsmar, FL.

For more information about Sagene Pharmaceuticals, please visit the Company’s website at www.sagenepharma.com.

About USF Health USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Biomedical Sciences and the School of Physical Therapy and Rehabilitation Sciences; and the USF Physician’s Group. The University of South Florida is a Top 50 research university in total research expenditures among both public and private institutions nationwide, according to the National Science Foundation. For more information, visit www.health.usf.edu

Media contact:
Anne DeLotto Baier, USF Health Communications
abaier@health.usf.edu or (813) 974-3303

The U.S. Food and Drug Administration yesterday designated fast-track status to EPI-743 for the treatment of Friedreich’s ataxia – a move that will help accelerate clinical development of the investigational drug, currently being tested in a multisite, double-blind, placebo-controlled trial led by the University of South Florida.

The phase 2b clinical trial of EPI-743 in adults with Friedreich’s ataxia, sponsored by Edison Pharmaceuticals, Inc., in collaboration with the Friedrich’s Ataxia Research Alliance, has been underway since early 2013 at the USF Health, the Children’s Hospital of Philadelphia and the University of California in Los Angeles.  Neurologist Theresa Zesiewicz, MD, director of the USF Ataxia Research Center, is the lead investigator for the national trial.

Researchers are primarily testing the effectiveness of EPI-743, a potent antioxidant, on vision, in patients with Friedreich’s ataxia, many of whom experience varying degrees of visual changes. Secondarily, the study is evaluating neurological function.

For more on the fast track status of EPI-743, go to: http://www.firstwordpharma.com/node/1195801#axzz2wKgT2a1W

First patient enrolled to evaluate investigational drug’s effectiveness  in rare Friedreich’s ataxia genotype

Mountain View, CA; Downingtown, PA; & Tampa, FL (Nov. 4, 2013) — Edison Pharmaceuticals, the Friedreich’s Ataxia Research Alliance (FARA) and the University of South Florida (USF) today announced the initiation of a phase 2 study entitled, “Phase 2A Clinical Trial of EPI-743 on Visual Function in Friedreich’s Ataxia Patients with Point Mutations.”

Given the rarity of the Friedreich’s ataxia point mutation genotype, the trial is a single-arm, subject-controlled study lasting three months, followed by a three-month extension phase.  Study participants must be between the ages of 18 and 65, possess genetic confirmation of Friedreich’s ataxia point mutation defect, and meet certain disease severity criteria. The primary endpoint of the trial is visual function, with secondary endpoints including neurological and neuromuscular function, and disease-relevant biomarkers.  More information on study specifics is available on ClinicalTrials.gov.

This trial will be conducted at USF Health – a member of the Collaborative Clinical Research Network in Friedreich Ataxia– and is funded by a grant awarded by the Friedreich’s Ataxia Research Alliance. The study sponsor is USF, and the principal investigator is Theresa A. Zesiewicz, MD, FAAN, professor of neurology and director of the USF Ataxia Research Center.

“FARA has been eager to see drug development efforts extend into ultra-rare subgroups of Friedreich’s ataxia, such as those harboring point mutations,” said FARA President Ronald Bartek. “This is the first study dedicated to examining safety and investigational drug response in adults with point mutations in the frataxin gene that represent approximately 2 to 4 percent of the Friedreich’s ataxia patients.”

EPI-743 is currently being evaluated in a phase 2B placebo-controlled Friedreich’s ataxia trial in individuals with the more common triplet repeat expansion defect in frataxin synthesis.

Friedreich’s Ataxia

Friedreich’s ataxia is an autosomal recessive nuclear DNA inherited mitochondrial disease, affecting an estimated 1 in 30,000 individuals in the United States and Europe. Friedreich’s ataxia is caused by a defect in the gene frataxin, which encodes a 210 amino acid protein that participates in iron-sulfur (Fe-S) cluster protein assembly. As the majority of these Fe-S cluster proteins are localized in the respiratory chain in the mitochondria, patients with Friedreich’s ataxia present with “energy failure” symptoms including ataxia, muscle weakness, heart failure, diabetes, and visual, speech, and hearing deficiencies. Friedreich’s ataxia is a highly debilitating and life-shortening disease and is a member of a larger family of diseases called mitochondrial disease that share as a common biochemical mechanism defects in cellular energy metabolism. There are no FDA-approved drugs for Friedreich’s ataxia.

EPI-743

EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of Friedreich’s ataxia and other inherited mitochondrial diseases.

Edison Pharmaceuticals

Edison Pharmaceuticals is a biotechnology company dedicated to developing treatments for children and adults with mitochondrial diseases. More information can be found at www.edisonpharma.com.

FARA

The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501©(3), non-profit, tax-exempt organization dedicated to curing Friedreich’s ataxia (FA) through research. FARA grants and activities provide support for basic and translational FA research, pharmaceutical/ biotech drug development, clinical trials, and scientific conferences. FARA also serves as a catalyst, between the public and scientific community, to create worldwide exchanges of information that drive medical advances. For more information about FARA, visit them online at www.curefa.org.

USF Health

USF Health is an integral part of the University of South Florida, a global research university ranked 50^th in the nation by the National Science Foundation for both federal and total research expenditures among all U.S. universities. USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Biomedical Sciences and the School of Physical Therapy and Rehabilitation Sciences; and the USF Physician’s Group. For more information, visit www.health.usf.edu.