Efficacy and costs of spinal muscular atrophy drugs
Jonathan J. Darrow, Monica Sharma, Mansa Shroff, Anita K. Wagner
Abstract
Evaluating the benefits, risks, and costs of two drugs to treat spinal muscular atrophy raises questions about the future of rare disease medicines.
Topics & Concepts
Spinal muscular atrophyMedicineAtrophyDiseaseIntensive care medicinePhysical medicine and rehabilitationPathologyNeurogenetic and Muscular Disorders ResearchRNA modifications and cancerRNA Interference and Gene Delivery