Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

Whether caused by Duchenne muscular dystrophy, aging or other degenerative conditions, muscle loss can make everyday activities -- like walking, climbing stairs or even standing up -- a daily ...

Duchenne Muscular Dystrophy (DMD) is a rare progressive disorder. People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become ...

About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Myotonic dystrophy type 1 incurs high healthcare costs, driven by age and comorbidities like cardiac and respiratory issues. Early detection and integrated management of multisystemic ...