Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene ...

It’s a long protein with numerous redundant coils (purple balls), and acts like a shock absorber during ... muscle fibers are easily damaged. Duchenne muscular dystrophy results from mutations in the ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

DMD is an X-linked recessive disorder, a genetic pattern that causes it to develop almost always in males. DMD is caused by ...

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy ... "We look forward to a collaborative ...

An experimental gene therapy for Duchenne muscular dystrophy has showed better-than-expected results in a three-patient trial, according to preliminary data presented by Cambridge, Massachusetts–based ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...

These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD ... Sarepta's gene therapy – like rivals from Pfizer and Solid Biosciences – codes for a shortened form of the ...