Oligonucleotides, Antisense [CS] class drugs

6 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.
    Medication Details

  • exondys 51- eteplirsen injection

    (Eteplirsen)
    Sarepta Therapeutics, Inc.
    Exondys 51 treats Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation amenable to exon 51 skipping. Approved under accelerated approval based on observed dystrophin increases; continued approval contingent on confirmatory trial results.
    Medication Details

  • qalsody - tofersen injection

    (Tofersen)
    Biogen Inc.
    Qalsody treats ALS (amyotrophic lateral sclerosis) in adults with a SOD1 gene mutation. Approved under accelerated approval based on reduction in plasma neurofilament light chain; continued approval may depend on confirmatory trial results.
    Medication Details

  • spinraza - nusinersen injection, solution

    (Nusinersen)
    Biogen Inc.
    SPINRAZA treats spinal muscular atrophy (SMA) in pediatric and adult patients.
    Medication Details

  • 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.
    Medication Details
    Drug Interactions

  • wainua - eplontersen injection, solution

    (Eplontersen)
    Astrazeneca Pharmaceuticals Lp
    Wainua treats polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults.
    Medication Details