FocusDrug Development

Efficacy and costs of spinal muscular atrophy drugs

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Science Translational Medicine  11 Nov 2020:
Vol. 12, Issue 569, eaay9648
DOI: 10.1126/scitranslmed.aay9648

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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.

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