Efficacy and costs of spinal muscular atrophy drugs.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 33177183.
- Also identified by 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.
Medical subject headings
- Muscular Atrophy, Spinal
- Pharmaceutical Preparations