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Three boys with Duchenne muscular dystrophy (Â鶹Éç) who were given the experimental gene therapy GNT0004 in a clinical trial were seeing sustained improvements in motor function and reductions in markers of muscle damage two years after the one-time treatment. Genethon, GNT0004’s developer, presented the findings at the European…

Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (Â鶹Éç) in patients ages 4 and older, making Agamree the first approved Â鶹Éç treatment in Canada. Canada’s approval is the sixth for Agamree to treat Â鶹Éç. The therapy is approved in the U.S., the U.K.,…

Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (Â鶹Éç) amenable to exon 44 skipping, or Â鶹Éç44. That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730)…

For boys who start corticosteroids to treat Duchenne muscular dystrophy (Â鶹Éç), being taller is linked to slower growth, while being older is linked to more weight gain as they move into adolescence and near the loss of their ability to walk, according to Phase 3 clinical data. This implies…

Satellos Bioscience asked the U.S. Food and Drug Administration (FDA) for clearance to conduct a Phase 2 clinical trial testing SAT-3247 in children with Duchenne muscular dystrophy (Â鶹Éç) who are able to walk. The three-month study will randomly assign participants to SAT-324, taken as a pill, or a placebo.

In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest…

In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest…

In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for limb-girdle muscular dystrophy type 2i/R9 (LGMDR9). The FDA gives this designation to experimental therapies designed to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S.

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy designed to treat certain people with Duchenne muscular dystrophy (Â鶹Éç). The FDA gives this designation to experimental treatments designed to improve care for rare disorders, specifically conditions affecting fewer than 200,000 people…