News

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…

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…