News

Every newborn in the United States should now be screened for Duchenne muscular dystrophy (DMD), according to a recommendation from the U.S. Department of Health and Human Services (HHS). This federal decision marks a turning point for rare disease advocacy, potentially ending the “diagnostic odyssey” that many families face when…

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 Therapeutics, to qualify for a future priority review benefit for another experimental treatment in its pipeline. “This designation is an important regulatory…

Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping therapy for people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, after a Phase 1/2 trial showed encouraging results. Data from the registrational expansion cohort of the…

In the Phase 3 HOPE-3 clinical trial, the experimental cell therapy deramiocel outperformed a placebo at improving arm function and heart health in people with Duchenne muscular dystrophy (DMD), according to top-line results announced by its developer, Capricor Therapeutics. Capricor has said it plans to use the…

Upsher-Smith Laboratories is launching a new corticosteroid treatment for people with Duchenne muscular dystrophy (DMD) in the U.S. ages 5 and older. The therapy, branded Kymbee, is an oral tablet formulation of deflazacort, a corticosteroid available for DMD patients under brand names including Emflaza. Patients taking Kymbee…

A regimen of three immune-suppressing medications may improve the effectiveness of gene therapy for people with Duchenne muscular dystrophy (DMD), allowing patients to receive treatment more than once and making it accessible to people who are currently ineligible, a mouse study showed. DMD is caused by mutations in…

A Phase 1/2 clinical trial testing SRP-1003, a treatment for myotonic dystrophy type 1 (DM1), is progressing as planned and continues to enroll patients at higher doses. The Phase 1/2 study (NCT06138743) is expected to enroll 78 adults, ages 18 to 65, who have a genetically confirmed diagnosis…

New findings from research in mice are challenging longstanding beliefs about the causes of Duchenne muscular dystrophy (DMD), with evidence showing that the genetic disease is marked by abnormalities in muscle stem cells during fetal development — indicating DMD may start in the womb. According to the researchers, these findings suggest…

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), removing its conditional approval for DMD patients who can’t walk and including new safety warnings and precautions in light of two deaths related to the…

Agamree (vamorolone), a type of corticosteroid approved to help preserve muscle function in people with Duchenne muscular dystrophy (DMD), appears to work as well as traditional corticosteroids at maintaining walking ability, but carries a lower risk of side effects such as broken bones, eye problems, and stunted growth.