Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in ...

Care hurdles now add to the disease burden faced by U.S. sickle cell patients, despite the promise of emerging options like gene therapy.

Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a ​late-stage trial to better control ammonia levels linked to ‌a rare inherited disorder.

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

A team of University of Kentucky researchers has uncovered a surprising clue in the battle against Alzheimer's disease that could help doctors predict, and ultimately prevent, a common side effect of ...