Groundbreaking gene therapy offers hope for a boy with fatal muscular dystrophy, potentially slowing disease progression and ...

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an impressive ...

Chronic muscle inflammation in Duchenne muscular dystrophy (DMD) is driven by both infiltrating and resident macrophages, the latter stimulated by CSF-1 produced by fibro/adipogenic progenitors.

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy (DMD), following positive data from the ...

Muscular dystrophy, as a category ... So in the context of Duchenne specifically, in the muscle supportive category, we've always had steroids as the hallmark of what we're doing, but now there ...

The PJ Nicholoff Steroid Protocol has been instrumental in educating medical professionals on the unique care considerations for patients with Duchenne on long-term steroids who may have ...

The 16-year-old’s infectious smile and easy charm have won over everyone from community members to politicians in both Indianapolis and Washington, D.C. But behind his charisma is a powerful cause: a ...

Oskoui, M., et al. (2025) Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus Report of the AAN Guidelines Subcommittee. Neurology . doi ...