that produces the dystrophin protein. The condition affects all muscles, including cardiac, skeletal, and smooth muscles, severely affecting cardiac and motor functions, with patients commonly ...

Both Becker muscular dystrophy (BMD) and DMD are caused by a problem in skeletal muscle producing dystrophin. Dystrophin is completely absent in DMD, whereas people with BMD have some, but it’s ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

Dystrophin and DGC are also lost in Duchenne ... and it results in the loss of both fat and skeletal muscle mass. Cancer patients who develop wasting usually respond more poorly to therapy and ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) technology to deliver a gene designed to produce a shortened form of the ...

"Our data confirmed we successfully restored full-length dystrophin in both the skeletal and heart muscles of mice with DMD, leading to significant improvements in their muscle health, strength ...

For non-ambulatory patients, the FDA granted accelerated approval to Elevidys based on expression of Elevidys micro-dystrophin in skeletal muscle. The Food and Drug Administration (FDA ...

(Reuters) -Drug developer PepGen said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in ...

The selection of ENTR-601-45 is based on robust in vitro exon skipping and dystrophin restoration observed in patient derived skeletal and cardiac muscle cells (Figure 1) as well as in vivo ...

“Modulating fast skeletal muscle contraction protects ... gene yielding unstable and/or dysfunctional dystrophin expression in muscles. Individuals with BMD, typically males, have ongoing ...