Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

The FDA and Gene Therapy for Duchenne Muscular Dystrophy

1 May 2024

Abstract excerpt

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.

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to CAR-T cell therapy research
Share to Muscle Physiology and Disorders
Share to Neurogenetic and Muscular Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 38691382
DOI: 10.1001/jama.2024.5613

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

The FDA and Gene Therapy for Duchenne Muscular DystrophyPMID 38691382Read ↗

Gene therapy for duchenne muscular dystrophy: expectations and challenges.Semantic proximity Read ↗

Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophySemantic proximity Read ↗

Advances in Duchenne muscular dystrophy gene therapySemantic proximity Read ↗

New and emerging pharmacotherapy for duchenne muscular dystrophy: a focus on synthetic therapeutics.Semantic proximity Read ↗

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in FocusSemantic proximity Read ↗

Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?Semantic proximity Read ↗

Duchenne muscular dystrophy: Drug development and regulatory considerations.Semantic proximity Read ↗

Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophySemantic proximity Read ↗

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical TrialsSemantic proximity Read ↗

Food and Drug Administration Lifts Clinical Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Linked to Patient DeathSemantic proximity Read ↗

Gene therapy prospects for Duchenne muscular dystrophy.Semantic proximity Read ↗

After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy TrialSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.