Margarida Azevedo, MSc,  —

Retrope, Inc., a privately held clinical-stage pharmaceutical company leading the advance of a revolutionary new unifying theory of aging and degeneration, has just opened patient enrolment for a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of oral drug RT001. The study aims to determine the drug’s safety, tolerability and pharmacokinetic profile,…

Gene-therapy specialist company, AAVLife, recently acquired a patent from the United States Patent and Trademark Office for an experimental treatment of cardiomyopathy caused by the genetic disorder, Friedreich’s ataxia, which affects the nervous system and causes movement problems. People with this condition develop impaired muscle coordination (ataxia) that worsens over time and commonly…

The Friedrich’s Ataxia Research Alliance (FARA), a non-profit organization that supports scientific research aimed at finding treatments and a cure for FA, will be the benefactor of charity biking event rideATAXIA, which will feature biking icon and star of the movie “The Ataxian“, Kyle Bryant. The 4th year of RideATAXIA…

Horizon Pharma has recently announced it initiated a Phase 3 study evaluating the Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia study (“STEADFAST”) of ACTIMMUNE (interferon gamma-1b) for the treatment of people with Friedreich’s Ataxia (FA). ACTIMMUNE (Interferon gamma 1-b) is a biologically manufactured protein similar to…

The Research Acceleration and Innovation Network (TRAIN) has invited nonprofit organization Friedreich’s Ataxia Research Alliance (FARA), which works nationwide to support research and find a cure for the rare neuromuscular disease Friedreich’s ataxia (FA), to join its network of organizations. The program was launched in 2005 by FasterCures and…

Specialty biopharmaceutical Horizon Pharma plc has been awarded Fast Track status from the U.S. Food and Drug Administration (FDA) for the company’s ACTIMMUNE (interferon gamma-1b) to be used in patients with Friedreich’s ataxia (FA). ACTIMMUNE is a biologically developed protein resembling one produced naturally by the body to prevent infection,…

In a recent article titled “Consensus clinical management guidelines for Friedreich ataxia” published in the journal Orphanet Journal of Rare Diseases, Louise Corben from the Children’s Research Institute, Australia and colleagues on behalf of the Clinical Management Guidelines Writing Group Murdoch reviewed the…

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…

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 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…