Sarep­ta's Ex­ondys 51 is not cost-ef­fec­tive, nor par­tic­u­lar­ly ben­e­fi­cial for DMD pa­tients — ICER

Par­ents with chil­dren suf­fer­ing from Duchenne mus­cu­lar dy­s­tro­phy (DMD) — a rare, pro­gres­sive mus­cle wast­ing dis­ease that dis­pro­por­tion­ate­ly af­fects young boys — cheered in 2016 when Sarep­ta …

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