This week’s New England Journal of Medicine reported results of the pivotal CENTAUR clinical trial; the USF Health ALS Clinic was one of only two sites in Florida for the study

Tampa, FL (Sept. 2, 2020) – Despite two approved medications and at least 100 clinical trials later, an urgent need for new treatments to improve the lives of patients with amyotrophic lateral sclerosis (ALS), a relentlessly progressive and fatal disease, continues to exist.

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This week’s New England Journal of Medicine published results of the highly anticipated CENTAUR clinical trial, and included among the study’s coinvestigators was Tuan Vu, MD, professor of neurology at the Morsani College of Medicine and director of the USF Health ALS Clinic.

CENTAUR was a 24-week randomized, double-blind trial Phase 2/3 trial of 137 adults with ALS conducted across 25 top medical centers in the U.S. through the Northeast ALS (NEALS) Consortium. USF Health was one of two Florida sites to evaluate the safety and effect on disease progression of Amylx Pharmaceuticals’ investigational neuroprotective therapy, known as AMX0035. The oral therapy combines two different medicines: sodium phenylbutyrate and taurursodiol.

Using the Revised ALS Functional Rating Scale (ALSFRS-R) to measure daily functions such as the ability to walk, dress independently, self-feed, speak and breathe, the CENTAUR investigators demonstrated that treatment with AMX0035 decreased the rate of decline in patients with ALS compared to those with ALS receiving a placebo. The therapy was generally well tolerated, with similar rates of adverse events reported in the AMX0035 and placebo groups. For more details on the CENTAUR trial, including the open label extension study, and next steps, click here. 

“ALS is likely the end result of several different pathological processes leading to the death of nerve cells in the brain and spinal cord that control voluntary muscle movements — so there is no one treatment solution. To combat this complex neurodegenerative disease we need to target more than one process at the same time,” Dr. Vu said.  “This investigational drug protects motor neurons in a different way than the two currently approved ALS medications (riluzole and edaravone). It was beneficial in slowing disease progression, allowing patients to remain functional for a longer period of time.”

Only two drugs to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, have been approved by the FDA over the last 25 years.

“CENTAUR met its prespecified primary outcome, showing a clinically meaningful and statistically significant treatment benefit on the ALSFRS-R, the most commonly used scale in clinics worldwide to measure function in ALS,” said Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General and assistant professor of physical medicine and rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.”

AMX0035 was the first investigational therapy to demonstrate statistically significant benefit on the prespecified primary outcome in patients with ALS since edaravone, an intravenous drug approved by the U.S. Food and Drug Administration (FDA) in 2017.  The first and only other commercially available drug to treat ALS, riluzole, was FDA approved 25 years ago.

The NEJM study authors concluded that sodium phenybutyrate/taurursodiol (AMX0035) resulted in slower progression of ALS over 24 weeks as measured by the ALSFRS-R total score, but recommended longer and larger trials to further evaluate the therapy’s effectiveness and safety.

Dr. Tuan Vu, professor of neurology, directs the USF Health ALS Clinic. Below: Dr. Vu performs an electromyography (EMG), one of the tests used to help diagnose ALS.

Zbigniew (Ziggy) Grajzer of Clearwater, Fla, was diagnosed with ALS in November 2019 and began treatment at the USF Health ALS Clinic in February 2020. He takes both edaravone and riluzole and remains hopeful about any steps forward to find new ALS treatments to improve quality of life and maintain independence.  While he was not enrolled in CENTAUR, Grajzer, 69, participates in other ALS clinical trials at the USF Health whenever he can. “It is the only way to find a cure,” he said, “if not in time for me, then to help others in the future.”

Grajzer said he feels fortunate that his ALS appears to be progressing slowly, but acknowledges the struggles of living with a disease that gradually robs an individual’s ability to control muscle movement. He has lost use of his left hand, his legs are weaker, and he tires easily, even walking from one room to another.  “I enjoyed going everywhere, to the beach and the mountains. Now most of what I do is around the house. But at least I can still move, speak, eat, breathe and think, and I’m grateful for that,” he said.

Zbigniew (Ziggy) Grajzer began treatment at the USF Health ALS Clinic earlier this year.

CENTAUR, the recipient of an ALS Accelerated Therapeutics grant, is supported by The ALS Association, ALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS Consortium, Healey Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge.

ABOUT USF HEALTH ALS CLINIC
The USF Health Amyotrophic Lateral Sclerosis (ALS) Clinic in Tampa, Fla., offers the latest treatment, clinical research opportunities, supportive care and educational resources to help patients, their families and caregivers manage the effects of ALS. The clinic has been recognized by The ALS Association as a Certified Treatment Center of Excellence.

About AMYOTROPHIC LATERAL SCLEROSIS (ALS)
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The vast majority of patients with ALS (>90%) have sporadic disease, showing no clear family history. Approximately 6000 people are diagnosed with ALS in the United States every year with an approximately similar number of deaths every year.

ABOUT AMX0035
AMX0035 is an investigational neuroprotective therapy designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

-Video by Allison Long, USF Health Communications and Marketing

A new University of South Florida preclinical study finds that the regenerative cell therapy boosts motor nerve cell survival by repairing the blood-spinal cord barrier

TAMPA, Fla. (April 2, 2019) — Transplantation of human bone marrow-derived endothelial progenitor cells (EPCs) into mice mimicking symptoms of amyotrophic lateral sclerosis (ALS) helped more motor neurons survive and slowed disease progression by repairing damage to the blood-spinal cord barrier (BSCB), University of South Florida researchers report.

The study was published March 27 in Scientific Reports, one of the Nature journals. The findings contribute to a growing body of work exploring cell therapy approaches to barrier repair in ALS and other neurodegenerative diseases.

Human bone marrow-derived endothelial progenitor cells in vitro

The progressive degeneration of nerve cells that control muscle movement (motor neurons) eventually leads to total paralysis and death from ALS. Each day, an average of 15 Americans are diagnosed with the disease, according to the ALS Association.

Damage to the barrier between the blood circulatory system and the central nervous system has been recognized as a key factor in the development of ALS. A breach in this protective wall opens the brain and spinal cord to immune/inflammatory cells and other potentially harmful substances circulating in peripheral blood. The cascade of biochemical events leading to ALS includes alterations of endothelial cells lining the inner surface of tiny blood vessels near damaged spinal cord motor neurons.

This latest study by lead author Svitlana Garbuzova-Davis, PhD, and colleagues at the USF Health Morsani College of Medicine’s Center of Excellence for Aging & Brain Repair, builds upon a previous study showing that human bone marrow-derived stem cells improved motor functions and nervous system conditions in symptomatic ALS mice by advancing barrier repair. However, in that earlier USF study the beneficial effect was delayed until several weeks after cell transplant and some severely damaged capillaries were detected even after a high-dose treatment. So in this study, the researchers tested whether human EPCs – cells harvested from bone marrow but more genetically similar to vascular endothelial cells than undifferentiated stem cells – would provide even better BSCB restoration.

Svitlana Garbuzova-Davis, PhD

ALS mice were intravenously administered a dose of human bone-marrow derived EPCs.  Four weeks after transplant, the results of the active cell treatment was compared against findings from two other groups of mice:  ALS mice receiving a media (saline) treatment and untreated healthy mice.

The symptomatic ALS mice receiving EPC treatments demonstrated significantly improved motor function, increased motor neuron survival and slower disease progression than their symptomatic counterparts injected with media. The researchers suggest that these benefits leading to BSCB repair may have been promoted by widespread attachment of EPCs to capillaries in the spinal cord. To support this proposal, they point to evidence of substantially restored capillaries, less capillary leakage, and re-establishment of structural support cells (perivascular astrocytes) that play a role in helping form a protective barrier in the spinal cord and brain.

Further research is needed to clearly define the mechanisms of EPC barrier repair.  But, the study authors conclude: “From a translational viewpoint, the initiation of cell treatment at the symptomatic disease stage offered robust restoration of BSCB integrity and shows promise as a future clinical therapy for ALS.”

The USF study was supported by a grant from the National Institute of Neurological Disorders and Stroke.

Article citation:
Svitlana Garbuzova-Davis, Crupa Kurien, Edward Haller, David J. Eve, Stephanie Navarro, George Steiner, Ajay Mahendrasah, Surafuale Hailu, Mohammed Khatib, Kayla J. Boccio, Cesario V. Borlongan, Harry R. Van Loveren, Stanley H. Appel and Paul R. Sanberg. Human Bone Marrow Endothelial Progenitor Cell Transplantation into Symptomatic ALS Mice Delays Disease Progression and Increases Motor Neuron Survival by Repairing Blood-Spinal Cord Barrier, Scientific Reports, March 27, 2019. https://doi.org/10.1038/s41598-019-41747-4.

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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

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

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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