The National Ataxia Foundation (NAF) has announced that Dr. Susan Perlman was recently awarded a Research Grant to build a web-based National Ataxia Database (NAD). Susan Perlman is a professor in the Department of Neurology at the David Geffen School of Medicine at UCLA…
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A study led Dr. Meritxell Teixidó and Prof. Ernest Giralt from the Institute for Research in Biomedicine, Barcelona, entitled “Applying the Retro-Enantio Approach to Obtain a Peptide Capable of Overcoming the Blood–Brain Barrier” was published in the Angewandte Chemie. The study highlighted the…
In a recent study entitled “Genome-Engineering Tools to Establish Accurate Reporter Cell Lines That Enable Identification of Therapeutic Strategies to Treat Friedreich’s Ataxia” a team of researchers described a new system that allows the identification of novel molecular pathways responsible for Friedreich’s Ataxia, as well as potential new…
A study published in The Journal of Biological Chemistry provides a detailed analysis on iron-sulfur cluster biogenesis in mammalian mitochondria based on a novel experimental system. The study is entitled “Fe – S cluster biogenesis in isolated mammalian mitochondria: coordinated use of per sulfide sulfur…
Earning the Pioneer SCA Translational Research Award from the National Ataxia Foundation, Thorsten Schmidt, PhD at University of Tubingen in Germany is developing a novel treatment for spinocerebellar ataxia type 3 (SCA3). His winning research, “Targeting the Intracellular Localization of Ataxin-3…
The National Ataxia Foundation has proudly announced that 23 promising studies regarding ataxia being conducted in the United States, United Kingdom, Belgium, Portugal, Mexico and Germany were awarded funding at the December 2014 NAF Board of Directors meeting for fiscal year 2015. Between these 23 new studies that will receive financial…
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Friedreich’s Ataxia Patients’ Clinical Characteristics Studies To Improve Translational Research
In a recent study entitled “Biological and clinical characteristics of the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS) cohort: a cross-sectional analysis of baseline data,” researchers describe the biological and clinical characteristics of patients with Friedreich’s Ataxia as a model of clinical outcomes to be…
Intrexon Corporation has made an offer for the total acquisition of Exemplar Genetics, which is a company dedicated to study the treatment of life-threatening conditions, such as Friedreich’s ataxia, heart disease, cancer, cystic fibrosis, cardiac arrhythmia, as well as neuromuscular and neurodegenerative disorders. The company provides transgenic…
Reata Pharmaceuticals, Inc. has announced that it has enrolled the first patient in a new Friedreich’s Ataxia clinical trial in collaboration with AbbVie, and the Friedreich’s Ataxia Research Alliance. In their currently recruiting phase 2/3 clinical trial, “RTA 408 Capsules in Patients with Friedreich’s Ataxia – MOXIe,”…
Vitamin B3, a water soluble vitamin essential to processing fat and proteins in the human body, may also prove important to treating Friedreich’s Ataxia. A currently recruiting clinical trial from Imperial College London is studying the “Effect of Nicotinamide [Vitamin B3] in Friedreich’s Ataxia”…
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