Efficacy and costs of spinal muscular atrophy drugs

Sci Transl Med. 2020 Nov 11;12(569):eaay9648. doi: 10.1126/scitranslmed.aay9648.

Authors

Jonathan J Darrow¹, Monica Sharma², Mansa Shroff³, Anita K Wagner^{4

5}

Affiliations

¹ Program on Regulation, Therapeutics, and Law, Brigham and Women's Hospital and Harvard Medical School, Boston, MA 02120, USA. [email protected].
² IP-Dhyaan LLP, Dwarka, New Delhi 110075, India.
³ Harvard T.H. Chan School of Public Health, Boston, MA 02115, USA.
⁴ Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA 02215, USA.
⁵ Harvard Pilgrim Health Care Inc., Wellesley, MA 02481, USA.

PMID: 33177183
DOI: 10.1126/scitranslmed.aay9648

Abstract

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

Copyright © 2020 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Humans
Muscular Atrophy, Spinal* / drug therapy
Pharmaceutical Preparations*

Substances

Pharmaceutical Preparations