FDA Approves New Treatment For Rare Duchenne Muscular Dystrophy Mutation by Pooja Shete on March 1, 2021 at 12:22 AM
Casimersen (Amondys 45, Sarepta Therapeutics) injection.
The prevalence of DMD worldwide is about 1 in 3600 boys, girls can also be affected in rare cases. Symptoms of DMD appear around age 3 years but worsen steadily over time. Mutations in the DMD genes lead to decrease in dystrophin, which is a protein that strengthen muscle fibers and protect them from injury as muscles contract and relax.
The FDA said in a press release that this mutation of the DMD gene is amenable to exon 45 skipping and this is the first approval of a targeted treatment for patients with the mutation. In about 8 percent of patients, DMD gene mutation that is amenable to exon 45 skipping is present.