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AMONDYS 45

updated 2mo ago

by $SRPT Sarepta Therapeutics, Inc.🔗 ChEMBL

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Oligonucleotide drug with a maximum clinical stage of Approval (across all indications), with an approval for duchenne muscular dystrophy and muscular dystrophy.

targets

Dystrophin pre-mRNA

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brand

AMONDYS 45

trade names

Amondys 45

generic

CASIMERSEN

MOA

12.1 Mechanism of Action Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping [see Clinical Studies ( 14 )] .

indication

1 INDICATIONS AND USAGE AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. ( 1 )

AMONDYS 45

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Trials studying AMONDYS 45