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”…
Not all clinical trials vital to understanding Friedreich’s ataxia involve new therapies under evaluation before being marketed as treatment. Ronald Crystal, MD, of Weill Medical College of Cornell University in New York is sponsoring an observational case control study for “Characterization of the Cardiac…
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…
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…
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