Spending on Targeted Therapies for Duchenne Muscular Dystrophy-Reference-Cited by-同舟云学术

Spending on Targeted Therapies for Duchenne Muscular Dystrophy

Published:2024-04-02 Issue:13 Volume:331 Page:1151
ISSN:0098-7484
Container-title:JAMA
language:en
Short-container-title:JAMA

Author:

Bendicksen Liam¹,Kesselheim Aaron S.¹,Rome Benjamin N.¹

Affiliation:

1. Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s Hospital, Boston, Massachusetts

Abstract

This study estimates public and private spending on genetically targeted treatments for Duchenne muscular dystrophy during years in which the drugs were marketed without completed confirmatory studies.

Publisher

American Medical Association (AMA)

Link

https://jamanetwork.com/journals/jama/articlepdf/2816420/jama_bendicksen_2024_ld_240012_1711564200.25351.pdf

Reference6 articles.

1. The regulatory repercussions of approving muscular dystrophy medications on the basis of limited evidence.;Bendicksen;Ann Intern Med,2023

2. Approving a problematic muscular dystrophy drug: implications for FDA policy.;Kesselheim;JAMA,2016

3. Timing of confirmatory trials for drugs granted accelerated approval based on surrogate measures from 2012 to 2021.;Deshmukh;JAMA Health Forum,2023

4. Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. Press release. Sarepta Therapeutics. October 30, 2023. Accessed February 5, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0

5. National Institutes of Health. Duchenne muscular dystrophy. Accessed January 16, 2024. https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy

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