Patients are often the best experts on their disease, but what about emergency situations? Columnist Patrick Moeschen shares ...

Limb-girdle muscular dystrophy (LGMD) describes a group of rare genetic conditions affecting the hip and shoulder girdle muscles. LGMD typically causes muscle weakness and deterioration and can ...

The FDA has designated the viral vector rAAVrh74, which Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses in its gene therapy ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, ...

Others are so weak they need to use a wheelchair. In its later stages, limb-girdle muscular dystrophy can cause serious heart problems. Usually this type of muscular dystrophy doesn't appear until ...

Figure 1: Physical map of the candidate region for the LGMD 2G locus, showing approximate locations of 40 genetic markers. Figure 3: DNA sequence electrophoregrams representing the two mutations ...

Sarepta Therapeutics’ gene therapy to treat limb girdle muscular dystrophy type 2E/R4 gets US FDA platform technology designation: Cambridge, Massachusetts Friday, June 6, 2025, ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

4-10 About Limb-Girdle Muscular Dystrophy (LGMD) Limb-girdle muscular dystrophy (LGMD) is a term for a group of diseases that cause progressive weakness and wasting of the muscles in the arms and ...

LANCASTER, Pa., Jan. 20, 2025 /PRNewswire/ -- On July 18, 2025, The Speak Foundation will host the fourth International Limb Girdle Muscular Dystrophy (LGMD) Conference at the J.W. Marriott ...

2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused by mutations in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.6 Those ...