Viltolarsen (Viltepso; NS Pharma) is the second FDA-approved targeted treatment for patients with Duchenne muscular dystrophy (DMD) and a confirmed mutation on the DMD gene amenable to exon 53 ...

A new study shows that a single injection of CRISPR-Gold, as the new delivery system is called, into mice with Duchenne muscular dystrophy led to an 18-times-higher correction rate and a two-fold ...

Avidity Biosciences granted FDA Fast Track designation for AOC 1044 for treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping. News release. Avidity Biosciences. April 24 ...

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

Gene Therapy for Duchenne Muscular Dystrophy ... Duchenne muscular dystrophy is characterized by a mutation that ... The treatment is contraindicated in patients with any deletion in exon 8 ...

Sarepta Therapeutics announces FDA approval of Amondys 45™ (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. [ press release ...

Avidity Biosciences Inc. has announced positive data in healthy volunteers from its ongoing phase 1/2 clinical trial of a proprietary monoclonal antibody to treat Duchenne muscular dystrophy ...

By skipping more than a single exon, this so-called DNA band-aid becomes applicable to between 80 and 90 percent of Duchenne muscular dystrophy patients, including the mutation found in dogs.

Genetic mutation could worsen heart function in Duchenne muscular dystrophy patients. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2020 / 11 / 201104085145.htm ...

Eight therapies are approved for Duchenne muscular dystrophy (DMD) in the U.S., and all have been approved since 2016. "It took 30 years from the discovery of the gene associated with Duchenne ...

Duchenne muscular dystrophy (DMD) ... Women who have the DMD gene mutation don't usually show symptoms, but they are carriers for the disorder and can potentially pass it on to their children.

'CRISPR-Gold' fixes Duchenne muscular dystrophy mutation in mice. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2017 / 10 / 171003202913.htm ...