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6 results

exondys 51
(eteplirsen)
Sarepta Therapeutics, Inc.
Usage: 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.
qalsody
(tofersen)
Biogen Inc.
Usage: 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.
spinraza
(Nusinersen)
Biogen
Usage: SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in both pediatric and adult patients.
tegsedi
(inotersen)
Akcea Therapeutics, Inc.
Usage: TEGSEDI is indicated for treating the polyneuropathy associated with hereditary transthyretin-mediated amyloidosis in adults.
vyondys 53
(golodirsen)
Sarepta Therapeutics, Inc.
Usage: 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.
wainua
(EPLONTERSEN)
AstraZeneca Pharmaceuticals LP
Usage: WAINUA is indicated for the treatment of polyneuropathy associated with hereditary transthyretin-mediated amyloidosis in adults.

Antisense Oligonucleotide class drugs

exondys 51

qalsody

spinraza

tegsedi

vyondys 53

wainua