Efficacy and costs of spinal muscular atrophy drugs.

Darrow, Jonathan J; Sharma, Monica; Shroff, Mansa; Wagner, Anita K · Sci Transl Med · 2020

review · Level V

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Abstract

Evaluating the benefits, risks, and costs of two drugs to treat spinal muscular atrophy raises questions about the future of rare disease medicines.

Medical subject headings