Eteplirsen (Exondys 51)

Eteplirsen (Exondys 51) is covered only for Duchenne muscular dystrophy with a documented DMD gene mutation amenable to exon 51 skipping and is not covered for other mutations or non‑approved indications. Initial approval (12 months) requires clinical DMD diagnosis and genetic confirmation of an exon 51‑amenable mutation; reauthorization (12 months) requires evidence of benefit (e.g., increased dystrophin or improved 6‑minute walk distance), with a recommended dose of 30 mg/kg once weekly.

"For the treatment of Duchenne muscular dystrophy in an individual who has a confirmed mutation of the Duchenne muscular dystrophy gene that is amenable to exon 51 skipping"

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