Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available. Among lifestyle factors, physical activity stands out as possibly ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
Knowing the precise mechanisms by which MYC drives muscle growth could prove instrumental in creating therapies that reduce muscle loss from aging, potentially improving independence, mobility and ...
Please provide your email address to receive an email when new articles are posted on . RGX-202 was safe and well-tolerated in all 12 patients at two different doses with no serious adverse events.
WATERTOWN, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, today announced ...
Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver ...
The FDA has cleared the company's investigational new drug application seeking to begin the Phase I/II FUNCTION-DMD trial.
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
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