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FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections.

作者信息

Mullard Asher

出版信息

Nat Rev Drug Discov. 2023 Aug;22(8):610. doi: 10.1038/d41573-023-00103-y.

DOI:10.1038/d41573-023-00103-y
PMID:37353665
Abstract
摘要

相似文献

[1]
FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections.

Nat Rev Drug Discov. 2023-8

[2]
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[3]
Exon Skipping Therapy.

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[4]
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[5]
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[6]
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[7]
The role of immunosuppression in gene- and cell-based treatments for duchenne muscular dystrophy.

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[8]
[Frontline studies on Duchenne muscular dystrophy treatment].

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[9]
Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients.

Hum Gene Ther. 2018-4-5

[10]
Recent developments in Duchenne muscular dystrophy: facts and numbers.

J Cachexia Sarcopenia Muscle. 2017-10

引用本文的文献

[1]
A population-based study of severe, less common comorbidities in Duchenne muscular dystrophy.

J Neurol. 2025-8-28

[2]
Beyond the Cut: Long-read sequencing reveals complex genomic and transcriptomic changes in AAV-CRISPR therapy for Duchenne Muscular Dystrophy.

bioRxiv. 2025-8-1

[3]
Viral and non-viral vectors in gene therapy: current state and clinical perspectives.

EBioMedicine. 2025-7-1

[4]
Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.

Mol Ther. 2025-6-4

[5]
Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies.

Degener Neurol Neuromuscul Dis. 2025-4-12

[6]
Polyplex Nanomicelle-Mediated Pgc-1α4 mRNA Delivery Via Hydrodynamic Limb Vein Injection Enhances Damage Resistance in Duchenne Muscular Dystrophy Mice.

Adv Sci (Weinh). 2025-4

[7]
Science and Society: Pathways to Equitable Access and Delivery of Genomics Medicine in Africa.

Curr Genet Med Rep. 2025

[8]
Gene therapy for genetic diseases: challenges and future directions.

MedComm (2020). 2025-2-13

[9]
Delivery of genetic medicines for muscular dystrophies.

Cell Rep Med. 2025-1-21

[10]
High mobility group box 1 (HMGB1) is a potential disease biomarker in cell and mouse models of Duchenne muscular dystrophy.

Biol Open. 2024-9-15

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