News

Voyager Therapeutics and MassBiologics To Collaborate In Treatments For MS, ALS, Parkinson’s, And Friedreichs Ataxia

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…

Converting Skin Cells into Sensory Neurons Could Offer Therapeutic Approach For Friedreich’s Ataxia

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…

Researchers Explore Non-diabetic Insulin Resistance in Friedreich’s Ataxia

Although Friedreich’s ataxia is a condition with a known cause (a genetic abnormality on chromosome 9 that depletes cells of frataxin protein), some cases of the disease throw clinicians a curve ball. Such was the case for one 48-year-old man with a known diagnosis of Friedreich’s ataxia. He was…

IFN-γ-1b Helps 12 Friedreich’s Ataxia Patients in Trial

Results are in for an open-label phase 2 clinical trial of interferon gamma (IFN-γ) treatment for Friedreich’s ataxia (FRDA) sponsored by Children’s Hospital of Philadelphia along with collaborators at the Friedreich’s Ataxia Research Alliance and Vidara Therapeutics Research Ltd. David Lynch, MD, PhD, served as the principal investigator. The…