Patients with Friedreich’s ataxia commonly have difficulty walking due to imbalance issues or trouble conducting daily activities due to reduced upper arm function. Theresa Zesiewicz, MD, FAAN, from the University of South Florida (USF) in Tampa, is seeking to the study the course of these pains and is sponsoring a…
News
Specialty biopharmaceutical company Horizon Pharma plc has released a business update on its primary care and orphan divisions, announcing that the U.S. Food and Drug Administration (FDA) has agreed on the primary endpoint for the phase 3 study dedicated to a potential Friedreich’s Ataxia (FA), called ACTIMMUNE.
A large, international clinical trial sponsored by European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS) is currently recruiting 650 patients with genetically confirmed Friedreich’s Ataxia to benefit future research efforts through data acquisition and validation. “Patient Registry of the European Friedreich’s Ataxia Consortium for…
A pilot study on the safety and effects of interferon gamma-1b (IFN-γ) in children with Friedreich ataxia entitled “Open-label pilot study of interferon gamma-1b in Friedreich ataxia” was published in Acta Neurologica Scandinavica by L. Seyer, part of Dr. D. R. Lynch’s group from the…
Friedreich’s ataxia patients are at a risk of developing foot drop deformity, or the loss of dorsiflexion and eversion (up-and-down movement), in the ankle joint. A clinical trial sponsored by Royal National Orthopedic Hospital NHS Trust in London is seeking to test head-to-head two methods of treating foot drop…
A new study on Friedreich’s Ataxia entitled “Sensitivity of Spatiotemporal Gait Parameters in Measuring Disease Severity in Friedreich Ataxia,” was recently published in the journal Cerebellum. Friedreich’s ataxia (FRDA) is an autosomal recessive disease, with gait ataxia being the main source of morbidity. Mobility…
A team of researchers from India examined for the first time population-based genetic information of nine Trinucleotide repeat disorders (TRDs). The study entitled “Molecular genetic analysis of trinucleotide repeat disorders (TRDs) in Indian population and application of repeat primed PCR” was recently published in the…
When testing new compounds to treat Friedreich’s ataxia (FA), it is vital to have a metric for determining the efficacy of these compounds. Some studies have suggested using the iron content of dentate nuclei in the cerebellum as a biomarker for determining the efficacy of siderophores and antioxidants in…
Biopharmaceutical company Shire, is sponsoring a clinical trial for adults with Friedreich’s ataxia (FA) and is currently enrolling participants. The phase 1 clinical trial is evaluating VP 20629 (indole-3-propionic acid) against a placebo to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of treatment in FA patients 18 to 45…
Cambridge, Massachusetts based gene therapy startup, Voyager Therapeutics, has announced it will strategically collaborate with of the University of Massachusetts Medical School (UMMS)’s MassBiologics unit to develop scalable process methods using current good manufacturing practices (cGMP) for manufacture of recombinant adeno-associated viral (rAAV) vector treatments for a range of…
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