Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Researchers suggest discovery of genetic link between ASD and DM1 opens up potential new therapeutic avenues for ASD.

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy ... DMD patients with DMD mutations amenable to exon 44 skipping ...

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy ... specifically target genetic mutations like Sarepta Therapeutics ...

for people living with Duchenne muscular dystrophy mutations a We recently compiled a list of the 12 Best Multibagger Stocks to Buy Right Now. In this article, we are going to take a look at where ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24 ... form of inherited deafness called DFNA50 caused by mutations in microRNA, by using a novel in vivo CRISPR ...

The FDA cleared Sarepta to start dosing in a first-in-human clinical trial testing the gene therapy SRP-9005 in people with ...

Scientists had discovered how to insert beneficial mutations—such as the gene for ... application would be for inherited diseases such as muscular dystrophy—which would most likely involve ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

New research has uncovered a genetic connection between autism spectrum disorder (ASD) and myotonic dystrophy type 1 (DM1), a rare neuromuscular disease.