Duchenne muscular dystrophy: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

• All targets
• DMD 9
• ATM 0
• SIRT1 0
• EGFR 0
• AMPK 0
• HDAC 0
• mTOR 0
• SGLT2 1
• TNF 0
• CFTR 1
• CTLA-4 0
• G6PD 0
• GLP-1 0
• HTT 1
• IL-23 0
• IL-6 0
• NF1 1
• NRF2 0
• PINK1 0
• PPAR 0
• SMN1 1
• STK11 0
• TTR 1
• YAP 0

EU-13 is a filter, not a ranking: it only narrows the list.

24 of 37+ institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Terminated

  A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

  NCT06241950· Sarepta Therapeutics, Inc.· Updated 6 Nov 2026

  “Study is being terminated due to a business decision.”Registry text, quoted unchanged. Source

• Withdrawn

  Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)

  NCT05558813· Assistance Publique - Hôpitaux de Paris· Updated 20 Nov 2025

  “Cancellation of financing by the financier.”Registry text, quoted unchanged. Source

• Terminated

  A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

  NCT03362502· Pfizer· Updated 15 Sept 2025

  “All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003”Registry text, quoted unchanged. Source

• Terminated

  A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

  NCT06597656· Sarepta Therapeutics, Inc.· Updated 4 Sept 2025

  “Study is being terminated due to a business decision.”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

  NCT06128564· Hoffmann-La Roche· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Single-Cell Analysis 2
• Organoids 3
• CRISPR-Cas Systems 13
• Gene Expression Profiling 7
• Sequence Analysis, RNA 1
• Whole Genome Sequencing 4
• Exome Sequencing 5
• Proteomics 13
• Metabolomics 1
• Mass Spectrometry 2
• Immunohistochemistry 3
• Magnetic Resonance Imaging 19
• Machine Learning 2
• Cohort Studies 8

See which targets of this disease your abstract mentions, and go to the people working on them.