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 ...

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 ...

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the ...

Additionally, skeletal MRI conducted on Part 1 patients indicated ... complementing the Company’s existing leadership in Duchenne muscular dystrophy and limb-girdle muscular dystrophies and ...

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is ...

Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle mass, due to mutations in the dystrophin gene. Without the corresponding functional protein ...