Antisense Oligonucleotide class drugs

4 results

  • exondys 51

    (eteplirsen)
    Sarepta Therapeutics, Inc.
    EXONDYS 51 is indicated for treating Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene suitable for exon 51 skipping. Its approval is based on observed increases in dystrophin in skeletal muscle, pending further verification of clinical benefit in ongoing trials.
    Medication Details

  • qalsody

    (tofersen)
    Biogen Inc.
    QALSODY is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults with a superoxide dismutase 1 (SOD1) gene mutation. Approval is based on reduced plasma neurofilament light chain (NfL) levels, with continued approval pending verification of clinical benefit from further trials.
    Medication Details

  • vyondys 53

    (golodirsen)
    Sarepta Therapeutics, Inc.
    VYONDYS 53 is indicated for treating Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation suitable for exon 53 skipping. Approval is based on increased dystrophin production in skeletal muscle, with continued approval dependent on clinical benefit verification in further trials.
    Medication Details
    Drug Interactions