Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early diagnosis, multidisciplinary care, and emerging treatments like gene therapy ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.

Muscular dystrophy is caused by a lack of certain proteins that are needed for muscle structure and function. Specifically, many of the types of MD are caused by a lack of a protein called dystrophin, ...

This results in chromatin structure loosening and uncontrolled expression of ... Becker muscular dystrophy (BMD) is an X-linked recessive disorder caused by dystrophin in-frame deletion in the DMD ...

The team believes that the structure was in use until the 8th century BCE, the middle of the period when the Kings of Judah ruled. A scarab seal from the 8th century BCE uncovered during the ...

It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the ...

of the Phase 2 FORWARD-53 study of WVE-N531 showed highly consistent, mean muscle content-adjusted dystrophin expression of 9.0% (range: 4.6-13.9%), best-in-class muscle delivery, multiple ...