Charles Moore,  —

The Friedreich’s Ataxia Research Alliance (FARA) and the UPenn Center for Orphan Disease Research are sponsoring fundraising events May 20 to mark Friedreich’s Ataxia Awareness Day — an annual celebration to call attention to the rare, debilitating and often life-shortening degenerative neuromuscular disorder. Since its formation in 1998, FARA, a national, public,…

The Friedreich’s Ataxia Research Alliance (FARA) and Livonia, Michigan-based law firm Foley, Baron, Metzger & Juip (FBMJ) have teamed up to sponsor a movie premiere event featuring the highly anticipated “Rogue One: A Star Wars Story.” Proceeds will be designated to support research toward finding treatments and…

Cambridge, Massachusetts-based clinical-stage gene therapy company Voyager Therapeutics will host an R&D Day from 8:30 to 11 a.m. Eastern Time on Friday, April 29,  in New York City. Voyager specializes in the development of treatments for fatal and debilitating diseases of the central nervous system (CNS) such as…

Bamboo Therapeutics, Inc., a biotechnology company focused on developing gene therapies for rare central nervous system and neuromuscular diseases, including Friedrich’s ataxia, announced that it has acquired the University of North Carolina (UNC) at Chapel Hill’s viral Vector Core gene therapy manufacturing facility. Financial details of the deal…

Originally established in 2001 by two patient organisations: Association Française contre les Myopathies (AFM) and the European Organisation for Rare Diseases (EURORDIS), EuroBioBank is a unique network of biobanks that stores and distributes quality DNA, cell and tissue samples for scientists conducting research on rare diseases (RDs), including neuromuscular…

The Minneapolis, Minnesota based National Ataxia Foundation and Ataxia organizations around the world have dedicated September 25, 2015 as the 16th annual International Ataxia Awareness Day (IAAD) help raise awareness about ataxia. Most people have likely never heard of ataxia, which refers to a group of rare and often fatal…

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…

A research team led by scientists at The Scripps Research Institute (TSRI) in La Jolla, California — one of the world’s largest independent, not-for-profit organizations focusing on research in the biomedical sciences — has discovered a simple method for converting  human skin cells into neurons that locate and transmit…