It is CLINICAL TRIALS AWARENESS day… C3 often gets asks, are there any clinical trials underway for LGMD2A (a form of calpainopathy)? Yes! One form of clinical trial, called a natural history study, is currently underway. This type of study
Sarepta Therapeutics adds LGMD2A to their pipeline
Today, Sarepta Therapeutics, Inc. announced that it has signed an agreement with Nationwide Children’s Hospital giving Sarepta the exclusive option to Nationwide’s gene therapy candidate to treat limb girdle muscular dystrophy type 2A (LGMD2A), a form of calpainopathy. This program