The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

His mum also expressed the importance of highlighting muscular dystrophy, which predominantly affects boys and causes muscle loss in the thighs and pelvis, extending to the arms. Emily added ...

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 ...

They say the truth is stranger than fiction, and seeing it brought into sharper focus through the lens of documentary filmmaking has given us some of our most unforgettable viewing experiences ...

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 ...

Pep­Gen is “tem­porar­i­ly paus­ing” a Phase 2 study of its ex­on 51 skip­ping drug for Duchenne mus­cu­lar dy­s­tro­phy, it said Tues­day.