Duchenne muscular dystrophy: who works on it

One card per institution. Pick a target or a country to narrow the list.

• 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 390 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 Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

  NCT05689164· Pfizer· Updated 12 Nov 2026

  “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 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

• Terminated

  Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

  NCT01247207· PTC Therapeutics· Updated 8 Oct 2026

  “Sponsor decision”Registry text, quoted unchanged. Source

• Terminated

  Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders

  NCT07254988· University of Copenhagen· Updated 16 Apr 2026

  “Terminated since further recruitment proved difficult”Registry text, quoted unchanged. Source

• Terminated

  A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

  NCT06079736· PepGen Inc· Updated 2 Dec 2025

  “PGN-EDO51 development terminated by Sponsor”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

  Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

  NCT05429372· Pfizer· Updated 21 Oct 2025

  “All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003”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

• Withdrawn

  A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

  NCT06833931· PepGen Inc· Updated 24 Jun 2025

  “Global PGN-EDO51 development voluntarily discontinued by Sponsor”Registry text, quoted unchanged. Source

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

• A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

  NCT05689164· Pfizer· Source

• A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy

  NCT07911995· Grünenthal GmbH· Source

• Duchenne Electronic Health Record Study

  NCT07609394· The Duchenne Registry· Source

• 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

• A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

  NCT06450639· Hoffmann-La Roche· Source

• Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials

  NCT07674758· Vanderbilt University Medical Center· Source

• CureDuchenne Link®: A Resource for Research

  NCT04972604· CureDuchenne· Source

• Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

  NCT04281485· Pfizer· Source

• A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

  NCT06138639· Solid Biosciences Inc.· Source

• The Duchenne Registry

  NCT02069756· The Duchenne Registry· 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

• Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.

  Dis Model Mech, Aug 2026

• Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblasts.

  Int J Mol Sci, Aug 2026

• Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.

  Genome Med, Jul 2026

• A novel strategy to enhance precise targeting of the RNA base editor mxABE.

  Yi Chuan, Jun 2026

• Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.

  Cells, May 2026

• CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.

  Mol Ther, Mar 2026

• CRISPR Gene Editing for Nucleotide Repeat Expansion Disorders: A Systematic Review of Preclinical and Clinical Evidence.

  Genet Test Mol Biomarkers, Mar 2026

• Functional and structural pathologies in skeletal muscle of a rat model of Duchenne muscular dystrophy.

  Skelet Muscle, Feb 2026

• Knock-out of specific DMD gene isoforms in the parental hESC line SA001 using CRISPR/Cas9.

  Stem Cell Res, Dec 2025

• Muscle satellite cell editing by LNP-CRISPR-Cas9 to resist muscle injury.

  Cell Rep, Dec 2025

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