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The FDA and Gene Therapy for Duchenne Muscular Dystrophy

This Viewpoint examines the appropriateness of FDA accelerated approval of novel gene therapies to treat boys with Duchenne muscular dystrophy following clinical trials with surrogate outcomes that did not demonstrate net benefits.

Tue May 28, 2024 19:06
JAMA

Tue May 28, 2024 19:06
Urgent Need for Regulatory Oversight of Human Cellular/Tissue–Based Products

This Viewpoint discusses the death of a patient caused by unregulated biological products and efforts to encourage federal government oversight of such products.

Tue May 28, 2024 19:06
Injury Prevention Science and Firearm Injury in Pediatric Health

In this issue of JAMA, Wolf et al found that during the period from 2019 to 2021, the US experienced the greatest increase in all-cause pediatric fatalities (aged 1-19 years) in more than 50 years, with increased mortality disproportionately resulting from preventable injuries. Importantly, the authors identified widening racial and ethnic disparities...

Tue May 28, 2024 19:06
Audio Highlights

Listen to the JAMA Editor’s Audio Summary for an overview and discussion of the important articles appearing in this week’s issue of JAMA.

Tue May 28, 2024 19:06
Racial and Ethnic Disparities in All-Cause and Cause-Specific Mortality Among US Youth

This cross-sectional study compares all-cause and cause-specific mortality trends and rates among youth from different races and ethnicities between 1999 and 2021.

Tue May 28, 2024 19:06

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