Duchenne muscular dystrophy: who works on it

One card per institution. Pick a target to see only the groups that work on it.

• 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

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

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-EF

  Syriac Education, Abbasid Times: Reclaiming the Place of Syriac Intellectual Training under Muslim Rulers (SEAT)

  Universita Degli Studi di Catania· Catania, Italy· EU contribution €209,483· until Feb 2029

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-ERC-POC

  Targeted Genetic Manipulation of Muscle Stem Cells in vivo Towards New Treatment Strategies Against Muscular Dystrophies (StemAAV)

  Institut Pasteur· Paris, France· EU contribution €150,000· until May 2028

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-EF

  SKeletal Muscle - REgulation of FORce and Maturation through Tubulin isotypes (SKM-REFORMAT)

  Universite Lyon 1 Claude Bernard· Villeurbanne Cedex, France· EU contribution €242,261· until Jan 2028

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-ERC-POC

  Exploiting Transcriptional Adaptation to Develop New Genetic Compensation-based Therapies (TA2TRSQ)

  Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften Ev· Munchen, Germany· EU contribution €150,000· until Feb 2027

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-GF

  Deciphering the role of protein S-palmitoylation in skeletal muscle physiopathology (MyoPALM)

  Universita Degli Studi di Padova· Padova, Italy· EU contribution €320,924· until Aug 2027· 2 countries

5 of 10 running projects, newest first. Listed amounts together: €16.5m.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

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

• Proteomics of the skeletal muscle dystrophin complex.

  Adv Clin Chem, Sept 2026

• Integrative multi-omics analysis reveals lipid/metabolite dysregulation and temporal decoupling in disease progression.

  Sci Rep, Sept 2026

• Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy.

  Sci Rep, Aug 2026

• The perijunctional zone is a molecularly distinct muscle subdomain altered in Duchenne muscular dystrophy.

  Nat Commun, May 2026

• ANXA11 suppression restores muscular function in the mdx mouse model of Duchenne muscular dystrophy (DMD).

  Nat Commun, May 2026

• Potential proteomic biomarkers for monitoring clinical studies in Duchenne/Becker muscular dystrophy.

  Expert Rev Proteomics, May 2026

• Identification of novel protein biomarkers correlating with both cardiac and skeletal muscle indices in duchenne muscular dystrophy.

  J Neuromuscul Dis, Apr 2026

• Identification of myokines associated with the pathological stress response in the mdx mouse model of Duchenne muscular dystrophy.

  J Neuromuscul Dis, Dec 2025

• Spatially Resolved Profiling of Compartmentalized Muscle and Brain Inflammation.

  Eur J Immunol, Dec 2025

• Proteomics-based evaluation of AAV dystrophin gene therapy outcomes in mdx skeletal muscle.

  JCI Insight, Nov 2025

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