Fox NewsFamily sees hope in gene therapy for son diagnosed with fatal neurodegenerative diseaseFox NewsJoseph Hann was diagnosed with CLN7 in March 2017, and shortly after he and his family relocated to Texas for a promising gene therapy trial. (Fox 4 … Continue reading
SooTodayHis sister is giving him stem cells, his girlfriend is giving him a year of her life (3 photos)SooTodaySince then his girlfriend Amanda Levar (also in tablet) has sacrificed her schooling to take care of him and his sister Jessica Catling (hold… Continue reading
Life Sciences Intellectual Property Review |
FDA OKs Nationwide Children’s Hospital’s IND for Sarepta’s microdystrophin gene therapy for DMD; shares up 2%
Seeking Alpha The FDA has signed off on Nationwide Children’s Hospital’s IND application seeking approval to begin a Phase 1/2a study of evaluating a new gene therapy candidate, AAVrh74.MHCK7.micro-Dystrophin, in patients with Duchenne muscular dystrophy (DMD) … Sarepta Therapeutics (SRPT): FDA Clears GALGT2 Gene Therapy Program – William BlairStreetInsider.com Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene …GlobeNewswire (press release) Sarepta Therapeutics And Nationwide Children’s Hospital Announce US Food And Drug Administration (FDA …Clinical Leader |
Genetic Literacy Project |
What’s the excitement over gene therapy?
Genetic Literacy Project Gene therapy has been in the news a lot of late as a potential cure for a variety of genetic diseases. The biggest breakthrough involved a unanimous backing from U.S. Food and Drug Administration’s advisory panel for a new gene therapy from Spark … |