Insmed’s (INSM) treatment of Duchenne muscular dystrophy, the single-stranded, non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) vector containing alpha-myosin-heavy-chain/murine muscle creatine kinase 7 (MHCK7) promoter and micro-dystrophin elements, was granted FDA orphan designation, according to a post to the agency’s site.
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