U.S., Feb. 12 -- ClinicalTrials.gov registry received information related to the study (NCT07402122) titled 'Registry for Duchenne and Becker Muscular Dystrophy' on Jan. 16.
Brief Summary: Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys. The established "standard of care" has improved prognosis; however, a causal therapy is not yet available. In 2024 and 2025, the first disease-modifying therapies were approved. These include Vamorolone (Agamree(R)) as a corticosteroid replacement with a more favorable side-effect profile for children aged four and older, and Givinostat (Duvyzat(R)) as a combination therapy with corticoste...