Clinical Trials for Duchenne
List of all researches such as gene therapy, exon skipping, reducing inflammation and improving muscle growth & protection therapies for duchenne muscular dystrophy.
The table below contains those trials currently enrolling Duchenne patients and involving active therapies, as of the date indicated.
Last updated: May 15, 2026
For a comprehensive overview, please visit ClinicalTrials.gov and EUclinicaltrials.eu. If you need assistance in understanding clinical trials, we recommend reading our blog post which provides detailed insights to help you navigate and interpret the available information effectively. – ‘How to Participate in Clinical Trials for Duchenne (DMD)?‘ –
Gene Therapy
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| Sarepta Therapeutics | SRP-9001-303 (Delandistrogene Moxeparvovec) | Phase 3 | Duchenne Muscular Dystrophy Non-ambulatory or Ambulatory 8 Years to 18 Years | USA (AR, CA, FL, IL, IA, MD, MA, MO, NY, NC, OH, PA, VA) AUS, BEL, DEU, HKG, ISR, ITA, JPN, KOR, ESP, SWE, TWN, GBR | NCT05881408 (ENVISION) |
| Sarepta Therapeutics | SRP-9001-401 (Delandistrogene Moxeparvovec) | Phase 4 | An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy | USA (AK, CO, DC, FL, IN, KS, MI, PA, TN, TX, VA) | NCT06270719 (ENDURE) |
| Sarepta Therapeutics | SRP-9001-103 (Delandistrogene Moxeparvovec) | Phase 1 | An Interventional Study Ambulatory and non-ambulatory Participants 2 Years to 18 Years | USA (CA, MO, OH, VA) | NCT04626674 (ENDEAVOR) |
| Sarepta Therapeutics | SRP-9001-104 (Delandistrogene Moxeparvovec) Imlifidase | Phase 1 | Ambulatory 4 Years to 9 Years (Child) | Spain | NCT06241950 |
| Sarepta Therapeutics | SRP-9001-105 (Delandistrogene Moxeparvovec) | Phase 1 | Duchenne Muscular Dystrophy Patients with pre-existing AAV Abs 4 Years to 8 Years | USA (MO, OH) | NCT06597656 (HORIZON) |
| Sarepta Therapeutics | SRP-9001-305 (Delandistrogene Moxeparvovec) | Phase 3 | A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION) | USA, BEL, GER, HONG KONG, IT, JP, SP, TH, UK | NCT05967351 |
| Sarepta Therapeutics | SRP-9001-402 (Delandistrogene Moxeparvovec) | Phase 4 | Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting | NA | NCT07542314 ENHANCE |
| Roche | Delandistrogene Moxeparvovec | Phase 2 | Duchenne Muscular Dystrophy Up to 3 Years Mutation between Exons 18 and 79 | BEL, DEU, FRA, ITA, ESP, GBR | NCT06128564 (ENVOL) |
| Genethon | GNT 0004 | Pivotal Phase | 5 patients aged 6 and 10 have been treated with GNT0004, 4 in France and 1 in the UK. | FR, UK | GNT-016-MDYF |
| RegenxBio | RGX-202 | Phase 1 & 2 & 3 | Duchenne Muscular Dystrophy 1 Year and older Ambulatory Mutation in Exons 18 and above | USA (AR, CA, CO, IL, TX, VA) | NCT05693142 NCT05683379 |
| Solid Biosciences | SGT-001 | Phase 1 & 2 | Duchenne Muscular Dystrophy 4 Years to 17 Years (Child) | USA (CA, FL) | NCT03368742 |
| Solid Biosciences | SGT-003 IMPACT DUCHENNE | Phase 3 | 7 Years to 11 Years (Child) Exclusion Criteria: Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or exons 42 to 45, inclusive, of the DMD gene as documented by a genetic report. | Australia, Canada | NCT07160634 |
| Solid Biosciences | SGT-003 INSPIRE DUCHENNE | Phase 3 | 0 Years to 17 Years (Child) Exclusion Criteria: Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or exons 42 to 45, inclusive, of the DMD gene as documented by a genetic report. | USA, Canada, Italy, United Kingdom | NCT06138639 |
| Belief BioMed | BBM-D101 | Phase 1 | Duchenne Muscular Dystrophy 4 Years to 8 Years | Chine (Shanghai) | NCT06641895 |
| Insmed | INS1201 | Phase 1 | Duchenne Muscular Dystrophy 2 Years to 4 Years Ambulatory Mutation between Exons 18 and 58 | USA (GA) | NCT06817382 |
| Shanghai Siponuoyin Biotechnology | SPOT-mRNA03 | Early Phase 1 | 2 Years to 6 Years (Ambulant Males) | China | NCT07188012 |
| Ultragenyx | UX810 | Early Phase 1 | NA | NA | NA |
| Generium | GNR-097 | Early Phase 1 | NA | Russia | NA |
| Kinea Bio | KNA-123 Midi-Dystrophin Gene Therapy | Pre-Clinical | NA | NA | NA |
Exon Skipping
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| NS Pharma | NS-089/NCNP-02 (Exon 44) | Phase 2 | Duchenne Muscular Dystrophy 4 Years to 14 years Ambulatory Amenable to exon 44 skipping | USA (CO, GA, IL, KS, OH, OR, PA, TX, VA) Canada, Japan, South Korea, Turkiye | NCT05996003 |
| NS Pharma | NS‐050/NCNP‐03 (Exon 50) | Phase 1 & 2 | Duchenne Muscular Dystrophy 4 Years to 14 years Ambulatory Amenable to exon 50 skipping | USA (CO, GA, IL, KS, OH, OR, PA, TX, VA) Canada, Japan, South Korea, Turkiye | NCT06053814 |
| NS Pharma | NS-051/NCNP-04 (Exon 51) | Pre-Clinical | NA | NA | NA |
Nationwide Children's Hospital | AAV9 U7snRNA (Exon 2 Duplications) | Phase 1 & 2 | 6 Months to 13 Years (Child) Confirmed duplication of exon 2 in the DMD gene | USA (OH) | NCT04240314 |
| Wave Life Sciences | WVE-N531 (Exon 53) | Phase 2 | 4 Years to 18 Years (Child, Adult) Amenable to exon 53 skipping | Jordan, United Kingdom | NCT04906460 NCT07209332 |
| Avidity Biosciences | AOC 1044 (Exon 44) | Phase 2 | 7 Years to 27 Years (Child, Adult) Amenable to exon 44 skipping | USA | NCT06244082 |
| Avidity Biosciences | AOC 1044 (Exon 44) | Phase 3 | 7 Years to 16 Years (Child) Amenable to exon 44 skipping | NA | NCT07587242 (SAFARI44) |
| Avidity Biosciences | AOC 1045 (Exon 45) | Pre-Clinical | NA | NA | NA |
| Dyne Therapeutics | DYNE-251 (Exon 51) | Phase 1 & 2 | 4 Years to 16 Years (Child) Amenable to exon 51 skipping | USA, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom | NCT05524883 |
| Dyne Therapeutics | Exon 44, 45, 53 | Pre-Clinical | NA | NA | NA |
| PepGen | PGN-EDO51 (Exon 51) | Phase 2 | 6 Years to 16 Years (Child) Amenable to exon 51 skipping | Canada | NCT06079736 |
| Entrada Therapeutics | ENTR-601-44 (Exon 44) | Phase 1 & 2 | 4 Years to 20 Years (Child, Adult) Amenable to exon 44 skipping | Belgium, Italy, Spain, United Kingdom | NCT07037862 |
| Entrada Therapeutics | ENTR-601-45 (Exon 45) | Phase 1 & 2 | 4 Years to 20 Years (Child, Adult) Amenable to exon 45 skipping | Belgium, Italy, Netherlands, Spain, United Kingdom | NCT07038824 |
| Entrada Therapeutics | ENTR-601-50 (Exon 50) | Pre-Clinical | NA | NA | NA |
| Entrada Therapeutics | ENTR-601-51 (Exon 51) | Pre-Clinical | NA | NA | NA |
| BioMarin Pharmaceutical | BMN 351 (Exon 51) | Phase 1 & 2 | 4 Years to 10 Years (Child) Amenable to exon 51 skipping | Italy, Netherlands, Spain, Turkiye, United Kingdom | NCT06280209 |
| BioMarin Pharmaceutical | BMN 351 (Exon 51) | Phase 2 | 4 Years and older (Child, Adult, Older Adult) Participants must have completed 351-201 | Italy, Netherlands, Spain, Turkiye, United Kingdom | NCT07573631 |
| Jiao Tong University | LE051 (Exon 51) | Early Phase 1 | 4 Years to 8 Years (Child) Amenable to exon 51 skipping | China | NCT06900049 |
| Sqy Therapeutics | SQY51 (Exon 51) | Phase 1 & 2 | 6 Years and older (Child, Adult, Older Adult) Amenable to exon 51 skipping | France | NCT05753462 |
Reducing Inflammation
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
Nationwide Children's Hospital | Prednisolone | Phase 4 | 1 Month to 30 Months (Child) | USA | NCT05412394 |
Children's National Research Institute | Canakinumab | Phase 1 & 2 | 2 Years and older (Child, Adult, Older Adult) | USA | NCT03936894 |
| Grünenthal GmbH | GRT6019 | Phase 1 | 18 Years to 55 Years (Adult) | USA | NCT07317063 |
Improving Muscle Growth & Protection
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| Capricor Therapeutics | CAP-1002 | Phase 3 | 10 Years and older (Child, Adult, Older Adult) | USA | NCT05126758 |
| Edgewise Therapeutics | EDG-5506 | Phase 2 | 4 Years to 17 Years (Child) | USA | NCT05540860 NCT06100887 NCT05540860 |
| Dystrogen Therapeutics | DT-DEC01 | Phase I and Phase II (Integrated) | Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing. | Poland | 2024-519004-27-00 |
| Satellos Bioscience | SAT-3247 | Phase 2 | 18 Years to 40 Years (Adult) | Australia | NCT06867107 |
| Satellos Bioscience | SAT-3247 | Phase 2A | 7 Years to 9 Years (Male Child) | USA, Australia, Canada, Serbia, United Kingdom | NCT07287189 |
| Myogenica | MyoPAXon | Phase 1 | 18 Years and older (Adult, Older Adult ) | USA | NCT06692426 |
| Keros Therapeutics | KER-065 | Phase 1 | In March 2025, the first top-line results from a Phase 1 clinical study of KER-065 in healthy volunteers were announced. KER-065 was generally well tolerated. | NA | NA |
MRNA - tRNA - saRNA Therapies
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| Ractigen Therapeutics | RAG-18 | Early Phase 1 | 4 Years to 15 Years (Male Child) | CHINA | NCT07282652 |
| Elixirgen Therapeutics | Bobcat mRNA | Pre-Clinical | NA | USA | NA |
| Tevard Biosciences | suptRNA | Pre-Clinical | NA | USA | NA |
Gene Editing
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| HuidaGene | HG302 | NA | Duchenne Muscular Dystrophy 4 Years to 8 Years Ambulatory Mutation in Exons 52, 52-61, or 52-63 | China | NCT06594094 |
| Precision BioSciences | PBGENE-DMD (Exon 45-55) | Phase 1/2a | 2 Years to 7 Years (Child) DMD mutation should fully contained between exons 45 to 55 [inclusive] | NA | NCT07429240 |
| Vertex Pharmaceuticals | CRISPR/Cas9 | Pre-Clinical | NA | NA | NA |
| MyoGene Bio | MyoDys (Exon 45-55) | Pre-Clinical | NA | NA | NA |
| GenAssist | GEN6050X (Exon 50) | Pre-Clinical | NA | NA | NA |
Regulating Calcium Balance
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| EspeRare Foundation | Rimeporide | Phase 1 & 2 | Rimeporide has the potential to be a first in class treatment to address cardiomyopathy in DMD patients | NA | NCT02710591 |
Improving Heart Function
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| ImmunoForge | Pemziviptadil | Phase 2 | NA | South Korea | NA |
Cumberland Pharmaceuticals | Ifetroban | Phase 2 | 7 Years and older (Child, Adult, Older Adult) | USA | NCT03340675 |
Medical University of Gdansk | Metoprolol | Phase 3 | 8 Years to 17 Years (Child) | Poland | NCT05066633 |
| Sardocor | SRD-001 | Phase 1 | 18 Years and older (Adult, Older Adult) Phase 2b to start soon | USA | NCT06224660 |
| Sardocor | SRD-003 | Phase 1/2a | Readout of Phase 1/2a expected in mid-2026 Phase 2b/3 to start in second half of 2026 | NA | NA |
| Secretome Therapeutics | STM-01 | Phase 1 | STM-01 is currently being evaluated in a Phase 1 non-Duchenne study of adults with Heart Failure With Preserved Ejection Fraction. | NA | NA |
Enhancing Bone Health
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| Roche/Genentech | Satralizumab | Phase 2 | 8 Years to 17 Years (Child) | USA, Italy, Poland, Spain | NCT06450639 |
Ultrasound-Mediated Delivery (UMD)
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| SonoThera | RIPPLE | Pre-Clinical | Using ultrasound-mediated delivery (UMD), SonoThera is developing a proprietary, nonviral, non-invasive approach which enables broad, highly-targeted biodistribution, delivery of diverse genetic payloads, in a redosable manner designed to be safe, well-tolerated and cost-effective. | NA | NA |
Other Major Studies
| Company | Drug | Phase | Indication & Inclusion Criteria | Locations | Clinical Trial |
|---|---|---|---|---|---|
| Tenaya Therapeutics | TN-301 | Pre-Clinical | HDAC Inhibitor | NA | NA |