Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

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 ...

Duchenne muscular dystrophy (DMD) is a genetic condition that primarily affects people assigned male at birth. However, due to the condition’s X-linked inheritance pattern, only people assigned ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

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 ...

Newborns in Minnesota can now be screened for Duchenne muscular dystrophy (DMD) and guanidinoacetate methyltransferase (GAMT) deficiency. While there is no cure, treatment can make symptoms and ...

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 ...

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