The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the ...

The Speak Foundation launched the LGMD Centers of Excellence, a network of clinics dedicated to improving care for people ...

Wheelchairs tend to be equated with disability, but columnist Patrick Moeschen sees them as tools that increase function and ...

Upsher-Smith launched Kymbee support websites offering guidance on treatment, access, and care for DMD patients, caregivers, ...

Share this page with email Share this page on Facebook Share this page on X Share this page on Reddit Print Preferred Source on Google Boys with Duchenne muscular dystrophy (DMD) may be able to regain ...

Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i ...

Share this page with email Share this page on Facebook Share this page on X Share this page on Reddit Print Preferred Source on Google As parents, we dedicate our lives to raising our children. We ...

When a patient is looking for a Duchenne specialist, I think it’s really good to check out Parent Project Muscular Dystrophy website. Also, in the Muscular Dystrophy Association website, they have a ...

Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), ...

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD), a step that could allow its developer, Atossa ...

The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec-rokl), ...

Duchenne muscular dystrophy (DMD) decreases muscle strength and mobility throughout the body, including in your mouth. Over time, these changes, along with challenges in maintaining daily hygiene, can ...