Active DMD Clinical Studies

How to Understand This DMD Active Studies

This page is a structured overview of ongoing Duchenne clinical trials in the DMD Warrior catalog. Unlike the main trial search, which lists every study with rich filters, this table focuses on active research grouped by therapeutic approach so you can scan sponsors, drugs, and phases in one view.

Studies are organized under headings such as Gene Therapy, Exon Skipping, mRNA / tRNA / saRNA Therapies, Gene Editing, and other standardized categories. Completed and terminated trials are excluded so the table reflects research that is still relevant for families exploring current options.

Each row shows the sponsoring company, investigational drug or product name, trial phase, variant or genotype requirements when listed, current recruitment status, countries with sites, and a link to the public trial page or registry identifier. Use the company and drug columns to compare programs within the same therapeutic approach.

Open a trial name or NCT ID to read full eligibility criteria, contact details, and links to ClinicalTrials.gov. Return to Home to filter by age, country, or status, visit DMD Clinical Trial Statistics for country and phase breakdowns, or explore the Duchenne Drug Development Pipeline for a horizontal phase view.

Last updated:September 1, 2026

Gene Therapy

Gene Therapy active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Sarepta Therapeutics	ELEVIDYS (ENHANCE)	Phase 4	Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Not Yet Recruiting	United States	NCT07542314
Genethon	GNT0004	Phase 3	Being included in the GNT-014-MDYF study Diagnosis of DMD based upon Gene testing positive with detailed genotyping except subjects with any mutations affecting exons 8 and/or 9	Actively Recruiting	Belgium, Spain, France	2023-505187-11-00
Sarepta Therapeutics	ELEVIDYS (ENVISION)	Phase 3	Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.	Active, Not Recruiting	United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom	NCT05881408
Solid Biosciences	SGT-003	Phase 3	Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.	Actively Recruiting	United States, Australia, Canada	NCT07160634
REGENXBIO	RGX-202	Phase 2/3	DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.	Actively Recruiting	United States, Canada	NCT05693142
GENERIUM	GNR-097	Phase 2	Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.	Actively Recruiting	Belarus, Russia	NCT07673809
Hoffmann-La Roche	ELEVIDYS (ENVOL)	Phase 2	Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test	Active, Not Recruiting	Belgium, France, Germany, Italy, Spain, United Kingdom	NCT06128564
Solid Biosciences	SGT-003	Phase 1/2	Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.	Actively Recruiting	United States, Canada, Italy, United Kingdom	NCT06138639
Solid Biosciences	SGT-001	Phase 1/2	Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.	Active, Not Recruiting	United States	NCT03368742
Insmed Gene Therapy LLC	INS1201	Phase 1	Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).	Actively Recruiting	United States	NCT06817382
Sarepta Therapeutics	ELEVIDYS	Phase 1	For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.	Actively Recruiting	United States	NCT04626674
Shanghai Jiao Tong University School of Medicine	BBM-D101	Early Phase 1	Genetically confirmed diagnosis of DMD.	Actively Recruiting	China	NCT06641895
Shanghai Siponuoyin Biotechnology Co Ltd	SPOT-mRNA03 (SPOT-03)	Early Phase 1	Boys aged ≥ 2 years to < 8 years and capable of walking independently for at least 10 meters.	Actively Recruiting	China	NCT07188012
Sarepta Therapeutics	SRP-9001-305 (Delandistrogene Moxeparvovec) ELEVIDYS	Interventional	The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received Elevidys in a previous clinical study. No study drug will be administered as part of this study.	Enrolling by Invitation	United States, Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom	NCT05967351
REGENXBIO	RGX-202	Observational	Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Actively Recruiting	United States	NCT05683379
Sarepta Therapeutics	SRP-9001-401 (Delandistrogene Moxeparvovec) ELEVIDYS (ENDURE)	Observational	Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Enrolling by Invitation	United States	NCT06270719

Sarepta Therapeutics

Not Yet Recruiting

Drug: ELEVIDYS (ENHANCE)
Phase: Phase 4
Variant Requirement: Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States
Clinical Trial: NCT07542314

Genethon

Actively Recruiting

Drug: GNT0004
Phase: Phase 3
Variant Requirement: Being included in the GNT-014-MDYF study Diagnosis of DMD based upon Gene testing positive with detailed genotyping except subjects with any mutations affecting exons 8 and/or 9
Locations: Belgium, Spain, France
Clinical Trial: 2023-505187-11-00

Sarepta Therapeutics

Active, Not Recruiting

Drug: ELEVIDYS (ENVISION)
Phase: Phase 3
Variant Requirement: Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Locations: United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom
Clinical Trial: NCT05881408

Solid Biosciences

Actively Recruiting

Drug: SGT-003
Phase: Phase 3
Variant Requirement: Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
Locations: United States, Australia, Canada
Clinical Trial: NCT07160634

REGENXBIO

Actively Recruiting

Drug: RGX-202
Phase: Phase 2/3
Variant Requirement: DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.
Locations: United States, Canada
Clinical Trial: NCT05693142

GENERIUM

Actively Recruiting

Drug: GNR-097
Phase: Phase 2
Variant Requirement: Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.
Locations: Belarus, Russia
Clinical Trial: NCT07673809

Hoffmann-La Roche

Active, Not Recruiting

Drug: ELEVIDYS (ENVOL)
Phase: Phase 2
Variant Requirement: Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
Locations: Belgium, France, Germany, Italy, Spain, United Kingdom
Clinical Trial: NCT06128564

Solid Biosciences

Actively Recruiting

Drug: SGT-003
Phase: Phase 1/2
Variant Requirement: Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
Locations: United States, Canada, Italy, United Kingdom
Clinical Trial: NCT06138639

Solid Biosciences

Active, Not Recruiting

Drug: SGT-001
Phase: Phase 1/2
Variant Requirement: Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
Locations: United States
Clinical Trial: NCT03368742

Insmed Gene Therapy LLC

Actively Recruiting

Drug: INS1201
Phase: Phase 1
Variant Requirement: Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).
Locations: United States
Clinical Trial: NCT06817382

Sarepta Therapeutics

Actively Recruiting

Drug: ELEVIDYS
Phase: Phase 1
Variant Requirement: For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Locations: United States
Clinical Trial: NCT04626674

Shanghai Jiao Tong University School of Medicine

Actively Recruiting

Drug: BBM-D101
Phase: Early Phase 1
Variant Requirement: Genetically confirmed diagnosis of DMD.
Locations: China
Clinical Trial: NCT06641895

Shanghai Siponuoyin Biotechnology Co Ltd

Actively Recruiting

Drug: SPOT-mRNA03 (SPOT-03)
Phase: Early Phase 1
Variant Requirement: Boys aged ≥ 2 years to < 8 years and capable of walking independently for at least 10 meters.
Locations: China
Clinical Trial: NCT07188012

Sarepta Therapeutics

Enrolling by Invitation

Drug: SRP-9001-305 (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Interventional
Variant Requirement: The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received Elevidys in a previous clinical study. No study drug will be administered as part of this study.
Locations: United States, Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom
Clinical Trial: NCT05967351

REGENXBIO

Actively Recruiting

Drug: RGX-202
Phase: Observational
Variant Requirement: Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States
Clinical Trial: NCT05683379

Sarepta Therapeutics

Enrolling by Invitation

Drug: SRP-9001-401 (Delandistrogene Moxeparvovec) ELEVIDYS (ENDURE)
Phase: Observational
Variant Requirement: Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States
Clinical Trial: NCT06270719

Exon Skipping

Exon Skipping active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Avidity Biosciences	AOC 1044 (SAFARI44)	Phase 3	Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping.	Actively Recruiting	Belgium, France, Germany, Italy, Spain, United Kingdom, United States	NCT07587242
Dyne Therapeutics	DYNE-251 (FORZETTO)	Phase 3	Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.	Actively Recruiting	United States	NCT07608432
Avidity Biosciences	AOC 1044 (EXPLORE44)	Phase 2	Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years. Confirmation of DMD gene mutation amenable to exon 44 skipping.	Active, Not Recruiting	United States	NCT06244082
BioMarin Pharmaceutical	BMN 351	Phase 2	Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study.	Enrolling by Invitation	Italy, Netherlands, Spain, Turkiye, United Kingdom	NCT07573631
Entrada Therapeutics	ELEVATE-LTE	Phase 2	Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201.	Not Yet Recruiting	Belgium, Italy, Netherlands, Spain, United Kingdom	NCT07682129
NS Pharma	Brogidirsen (NS-089/NCNP-02-201)	Phase 2	Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame.	Actively Recruiting	United States, Australia, Canada, Japan, New Zealand, South Korea, Turkiye	NCT05996003
Wave Life Sciences	WVE-N531 (FORWARD-53)	Phase 2	This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531. All patients will have rolled over from a previous study of WVE-N531.	Enrolling by Invitation	Jordan, United Kingdom	NCT07209332
BioMarin Pharmaceutical	BMN 351	Phase 1/2	Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping	Active, Not Recruiting	Italy, Netherlands, Spain, Turkiye, United Kingdom	NCT06280209
Dyne Therapeutics	DYNE-251 (DELIVER)	Phase 1/2	Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.	Active, Not Recruiting	United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom	NCT05524883
Entrada Therapeutics	ENTR-601-44 (ELEVATE-44)	Phase 1/2	Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.	Actively Recruiting	Belgium, Italy, Spain, United Kingdom	NCT07037862
Entrada Therapeutics	ENTR-601-45 (ELEVATE-45)	Phase 1/2	Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.	Actively Recruiting	Belgium, Italy, Netherlands, Spain, United Kingdom	NCT07038824
NS Pharma	NS-050/NCNP-03 (Meteor50)	Phase 1/2	Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame.	Active, Not Recruiting	United States, Canada, Japan, South Korea, Turkiye	NCT06053814
Wave Life Sciences	WVE-N531 (FORWARD-53)	Phase 1/2	Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention.	Actively Recruiting	United States, Jordan, United Kingdom	NCT04906460
GenAssist Therapeutics	GEN6050X	Early Phase 1	Patients with DMD gene exon deletion types confirmed by molecular diagnosis: 8-49, 20-49, 22-49, 51, 51-53, 51-55, 51-57, 51-59, 51-60, 51-67, 51-69, 51-75 or 51-78 and other mutations amenable to exon 50 skipping.	Active, Not Recruiting	China	NCT06392724
Shanghai Jiao Tong University School of Medicine	LE051	Early Phase 1	DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No.51.	Actively Recruiting	China	NCT06900049

Avidity Biosciences

Actively Recruiting

Drug: AOC 1044 (SAFARI44)
Phase: Phase 3
Variant Requirement: Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping.
Locations: Belgium, France, Germany, Italy, Spain, United Kingdom, United States
Clinical Trial: NCT07587242

Dyne Therapeutics

Actively Recruiting

Drug: DYNE-251 (FORZETTO)
Phase: Phase 3
Variant Requirement: Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
Locations: United States
Clinical Trial: NCT07608432

Avidity Biosciences

Active, Not Recruiting

Drug: AOC 1044 (EXPLORE44)
Phase: Phase 2
Variant Requirement: Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years. Confirmation of DMD gene mutation amenable to exon 44 skipping.
Locations: United States
Clinical Trial: NCT06244082

BioMarin Pharmaceutical

Enrolling by Invitation

Drug: BMN 351
Phase: Phase 2
Variant Requirement: Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study.
Locations: Italy, Netherlands, Spain, Turkiye, United Kingdom
Clinical Trial: NCT07573631

Entrada Therapeutics

Not Yet Recruiting

Drug: ELEVATE-LTE
Phase: Phase 2
Variant Requirement: Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201.
Locations: Belgium, Italy, Netherlands, Spain, United Kingdom
Clinical Trial: NCT07682129

NS Pharma

Actively Recruiting

Drug: Brogidirsen (NS-089/NCNP-02-201)
Phase: Phase 2
Variant Requirement: Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame.
Locations: United States, Australia, Canada, Japan, New Zealand, South Korea, Turkiye
Clinical Trial: NCT05996003

Wave Life Sciences

Enrolling by Invitation

Drug: WVE-N531 (FORWARD-53)
Phase: Phase 2
Variant Requirement: This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531. All patients will have rolled over from a previous study of WVE-N531.
Locations: Jordan, United Kingdom
Clinical Trial: NCT07209332

BioMarin Pharmaceutical

Active, Not Recruiting

Drug: BMN 351
Phase: Phase 1/2
Variant Requirement: Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping
Locations: Italy, Netherlands, Spain, Turkiye, United Kingdom
Clinical Trial: NCT06280209

Dyne Therapeutics

Active, Not Recruiting

Drug: DYNE-251 (DELIVER)
Phase: Phase 1/2
Variant Requirement: Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
Locations: United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom
Clinical Trial: NCT05524883

Entrada Therapeutics

Actively Recruiting

Drug: ENTR-601-44 (ELEVATE-44)
Phase: Phase 1/2
Variant Requirement: Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.
Locations: Belgium, Italy, Spain, United Kingdom
Clinical Trial: NCT07037862

Entrada Therapeutics

Actively Recruiting

Drug: ENTR-601-45 (ELEVATE-45)
Phase: Phase 1/2
Variant Requirement: Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.
Locations: Belgium, Italy, Netherlands, Spain, United Kingdom
Clinical Trial: NCT07038824

NS Pharma

Active, Not Recruiting

Drug: NS-050/NCNP-03 (Meteor50)
Phase: Phase 1/2
Variant Requirement: Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame.
Locations: United States, Canada, Japan, South Korea, Turkiye
Clinical Trial: NCT06053814

Wave Life Sciences

Actively Recruiting

Drug: WVE-N531 (FORWARD-53)
Phase: Phase 1/2
Variant Requirement: Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention.
Locations: United States, Jordan, United Kingdom
Clinical Trial: NCT04906460

GenAssist Therapeutics

Active, Not Recruiting

Drug: GEN6050X
Phase: Early Phase 1
Variant Requirement: Patients with DMD gene exon deletion types confirmed by molecular diagnosis: 8-49, 20-49, 22-49, 51, 51-53, 51-55, 51-57, 51-59, 51-60, 51-67, 51-69, 51-75 or 51-78 and other mutations amenable to exon 50 skipping.
Locations: China
Clinical Trial: NCT06392724

Shanghai Jiao Tong University School of Medicine

Actively Recruiting

Drug: LE051
Phase: Early Phase 1
Variant Requirement: DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No.51.
Locations: China
Clinical Trial: NCT06900049

mRNA / tRNA / saRNA Therapies

mRNA / tRNA / saRNA Therapies active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Peking Union Medical College Hospital	RAG-18	Early Phase 1	Male patient with Duchenne Muscular Dystrophy (DMD), able to provide a written diagnosis from a specialist and a verifiable genetic test report.	Active, Not Recruiting	China	NCT07282652

Peking Union Medical College Hospital

Active, Not Recruiting

Drug: RAG-18
Phase: Early Phase 1
Variant Requirement: Male patient with Duchenne Muscular Dystrophy (DMD), able to provide a written diagnosis from a specialist and a verifiable genetic test report.
Locations: China
Clinical Trial: NCT07282652

Gene Editing

Gene Editing active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Precision BioSciences	PBGENE-DMD	Phase 1/2	Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive])	Actively Recruiting	United States	NCT07429240

Precision BioSciences

Actively Recruiting

Drug: PBGENE-DMD
Phase: Phase 1/2
Variant Requirement: Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive])
Locations: United States
Clinical Trial: NCT07429240

Improving Muscle Growth & Protection

Improving Muscle Growth & Protection active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Capricor	Deramiocel (HOPE-3)	Phase 3	Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator.	Active, Not Recruiting	United States	NCT05126758
Capricor	Deramiocel (HOPE-2-OLE)	Phase 2	Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12.	Active, Not Recruiting	United States	NCT04428476
Edgewise Therapeutics	Sevasemten (EDG-5506)	Phase 2	A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.	Active, Not Recruiting	United States	NCT05540860
Edgewise Therapeutics	Sevasemten (EDG-5506)	Phase 2	Aged 6 to 17 with a documented mutation on the DMD gene and phenotype consistent with DMD. Prior receipt of an AAV-based gene therapy (≥ 2 years after documented receipt of gene therapy administration or ≥ 3 years after randomization in a randomized study).	Active, Not Recruiting	United States	NCT06100887
Satellos Bioscience	SAT-3247	Phase 2	Previously participated in the SAT-3247-CL-101 parent clinical trials.	Enrolling by Invitation	Australia	NCT06867107
Satellos Bioscience	SAT-3247	Phase 2	Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene. Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening.	Actively Recruiting	United States, Australia, Belgium, Canada, Poland, Serbia, Spain, United Kingdom	NCT07287189
Dystrogen Therapeutics	DT-DEC01	Phase 1/2	Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing.	Actively Recruiting	Poland	2024-519004-27-00
Myogenica	MyoPAXon	Phase 1	Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy.	Actively Recruiting	United States	NCT06692426

Capricor

Active, Not Recruiting

Drug: Deramiocel (HOPE-3)
Phase: Phase 3
Variant Requirement: Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator.
Locations: United States
Clinical Trial: NCT05126758

Capricor

Active, Not Recruiting

Drug: Deramiocel (HOPE-2-OLE)
Phase: Phase 2
Variant Requirement: Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12.
Locations: United States
Clinical Trial: NCT04428476

Edgewise Therapeutics

Active, Not Recruiting

Drug: Sevasemten (EDG-5506)
Phase: Phase 2
Variant Requirement: A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
Locations: United States
Clinical Trial: NCT05540860

Edgewise Therapeutics

Active, Not Recruiting

Drug: Sevasemten (EDG-5506)
Phase: Phase 2
Variant Requirement: Aged 6 to 17 with a documented mutation on the DMD gene and phenotype consistent with DMD. Prior receipt of an AAV-based gene therapy (≥ 2 years after documented receipt of gene therapy administration or ≥ 3 years after randomization in a randomized study).
Locations: United States
Clinical Trial: NCT06100887

Satellos Bioscience

Enrolling by Invitation

Drug: SAT-3247
Phase: Phase 2
Variant Requirement: Previously participated in the SAT-3247-CL-101 parent clinical trials.
Locations: Australia
Clinical Trial: NCT06867107

Satellos Bioscience

Actively Recruiting

Drug: SAT-3247
Phase: Phase 2
Variant Requirement: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene. Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening.
Locations: United States, Australia, Belgium, Canada, Poland, Serbia, Spain, United Kingdom
Clinical Trial: NCT07287189

Dystrogen Therapeutics

Actively Recruiting

Drug: DT-DEC01
Phase: Phase 1/2
Variant Requirement: Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing.
Locations: Poland
Clinical Trial: 2024-519004-27-00

Myogenica

Actively Recruiting

Drug: MyoPAXon
Phase: Phase 1
Variant Requirement: Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy.
Locations: United States
Clinical Trial: NCT06692426

Reducing Inflammation

Reducing Inflammation active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Nationwide Children's Hospital	Prednisolone	Phase 4	Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.	Actively Recruiting	United States	NCT05412394
Catalyst Pharmaceuticals	Vamorolone (AGAMREE)	Observational	Patient has a current, active prescription for, or is on, AGAMREE.	Actively Recruiting	United States, Puerto Rico	NCT06564974

Nationwide Children's Hospital

Actively Recruiting

Drug: Prednisolone
Phase: Phase 4
Variant Requirement: Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
Locations: United States
Clinical Trial: NCT05412394

Catalyst Pharmaceuticals

Actively Recruiting

Drug: Vamorolone (AGAMREE)
Phase: Observational
Variant Requirement: Patient has a current, active prescription for, or is on, AGAMREE.
Locations: United States, Puerto Rico
Clinical Trial: NCT06564974

Improving Heart Function

Improving Heart Function active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Sardocor	SRD-001	Phase 1	Diagnosis of DMD with confirmatory genetic testing.	Actively Recruiting	United States	NCT06224660

Sardocor

Actively Recruiting

Drug: SRD-001
Phase: Phase 1
Variant Requirement: Diagnosis of DMD with confirmatory genetic testing.
Locations: United States
Clinical Trial: NCT06224660

Enhancing Bone Health

Enhancing Bone Health active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Hoffmann-La Roche	Satralizumab	Phase 2	A definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Active, Not Recruiting	United States, Denmark, Italy, Poland, Spain, Ukraine	NCT06450639

Hoffmann-La Roche

Active, Not Recruiting

Drug: Satralizumab
Phase: Phase 2
Variant Requirement: A definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States, Denmark, Italy, Poland, Spain, Ukraine
Clinical Trial: NCT06450639

Other Therapeutic Approaches

Other Therapeutic Approaches active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Keros Therapeutics	KER-065	Phase 2	Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test.	Not Yet Recruiting	United States	NCT07704099
MED Institute Inc.	UC-MSC	Phase 2	Male sex by birth with a genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD).	Not Yet Recruiting	United States	NCT06579352

Keros Therapeutics

Not Yet Recruiting

Drug: KER-065
Phase: Phase 2
Variant Requirement: Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test.
Locations: United States
Clinical Trial: NCT07704099

MED Institute Inc.

Not Yet Recruiting

Drug: UC-MSC
Phase: Phase 2
Variant Requirement: Male sex by birth with a genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD).
Locations: United States
Clinical Trial: NCT06579352