Increased Protein Synthesis [PE] class drugs

4 results
  • amondys 45- casimersen injection

    (Casimersen)
    Sarepta Therapeutics, Inc.
    AMONDYS 45 treats Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation amenable to exon 45 skipping. Approved under accelerated approval based on increased dystrophin production in skeletal muscle; continued approval contingent on confirmatory trial results.
  • spinraza - nusinersen injection, solution

    (Nusinersen)
    Biogen Inc.
    SPINRAZA treats spinal muscular atrophy (SMA) in pediatric and adult patients.
  • vyondys 53- golodirsen injection

    (Golodirsen)
    Sarepta Therapeutics, Inc.
    Vyondys 53 treats Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation amenable to exon 53 skipping. Approved under accelerated approval based on increased dystrophin production in skeletal muscle; continued approval may depend on confirmatory trial results.