The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne muscular ...

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Add Yahoo as a preferred source to see more of our stories on Google. Elevidys has been approved for use in ambulatory patients aged four years and older who have confirmed mutations in the DMD gene.

The trial aims to assess the tolerability, safety, efficacy, functional outcomes, and dystrophin expression in affected patients.

With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne ...

The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 years and ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

Certain genetic mutations in patients with DMD influence the timing of ambulation loss, with exon 44 skipping, exons 3 through 7 deletions, and exon 2 duplications linked to delayed loss. The study's ...

Duchenne muscular dystrophy (DMD) is a genetic condition caused by a change, or mutation, in the DMD gene. This gene normally tells the body how to make dystrophin, a protein that helps protect ...

Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein. The combination of gene editing and patient-specific stem cells may offer a new ray of ...

SAN DIEGO, July 23, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide ...