It’s only fair to share… FDA approves first drug for spinal muscular atrophy New therapy addresses unmet medical need for rare disease The U.S. Food and Drug Administration today approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle …
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FDA grants accelerated approval to first drug for Duchenne muscular dystrophy
It’s only fair to share… CAS 1173755-55-9 eteplirsen, eteplirsén [Spanish], étéplirsen [French] , eteplirsenum [Latin], этеплирсен [Russian], إيتيبليرسان [Arabic] Structure credit http://lgmpharma.com/eteplirsen-still-proves-efficacious-duchenne-drug/ FDA grants accelerated approval to first drug for Duchenne muscular dystrophy New therapy addresses unmet medical need The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy …