Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population.

doi:10.3390/genes17070826

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

Community

Back to search

Article

Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population.

Genes - 20 Jul 2026

Khaidarov Saken, Moldakaryzova Aizhan, Dautov Dias, Sagatbayeva Nurgul, Yeszhan Banu, Nurgaliyeva Bayan, Kenbayeva Saltanat, Abdullayeva Gulban, Rabandiyarov Marat, Tazhibayeva Karlygash N, Sadykova Assel, Yermekbay Aibek, Aidarov Askar, Aidarov Daulet, Aidarova Aray, Kurbaniyazova Saniyam, Abiyrova Nazym, Kulmaganbetov Mukhit

Abstract excerpt

BACKGROUND: Duchenne muscular dystrophy (DMD) has, for the first time, several mutation-specific drugs in clinical use. Four exon-skipping antisense oligonucleotides and one adeno-associated virus (AAV) micro-dystrophin gene therapy carry US FDA approval, and one nonsense-readthrough agent (ataluren) holds a conditional European authorisation that has since been placed under review; ataluren has never been...

Read the complete abstract on PubMed

Topics

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

Share to Dependovirus

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Dystrophin

Share to Genetic Therapy

Share to Morpholinos

Share to Muscular Dystrophy, Duchenne

Share to Oligonucleotides

Share to Oligonucleotides, Antisense

Share to Oxadiazoles

Source

Open in PubMed Open full text

Identifiers

PMID: 42510866
DOI: 10.3390/genes17070826
PMCID: PMC13410084

Genes

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population.PMID 42510866Read ↗

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United StatesSemantic proximity Read ↗

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

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 TrialSemantic proximity Read ↗

From Bench to Bedside: A Meta-Analysis of Translational Research in Duchenne Muscular DystrophySemantic proximity Read ↗

Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular DystrophySemantic proximity Read ↗

DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.Semantic proximity Read ↗

Delandistrogene Moxeparvovec: First Approval.Semantic proximity Read ↗

Landscape and Heterogeneity of Treatment Outcome Measures in Facioscapulohumeral Muscular Dystrophy: A Systematic Literature ReviewSemantic proximity Read ↗

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE RegistrySemantic proximity Read ↗

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefitsSemantic proximity Read ↗

Life Expectancy in Duchenne Muscular Dystrophy: A Systematic Review and Reconstructed Individual Patient Data Meta-AnalysisSemantic proximity Read ↗

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophySemantic proximity Read ↗

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