Duchenne Muscular Dystrophy

Rare Diseases
47
Pipeline Programs
20
Companies
50
Clinical Trials
6 recruiting
3
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
7
10
22
0
5
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
350%
RNA Therapeutic
117%
Cell Therapy
117%
Monoclonal Antibody
117%
+ 46 programs with unclassified modality

On Market (3)

Approved therapies currently available

PTC Therapeutics
EMFLAZAApproved
deflazacort
PTC Therapeutics
Corticosteroid [EPC]oral2017
U
JAYTHARIApproved
deflazacort
Unknown Company
Corticosteroid [EPC]oral2025
U
PYQUVIApproved
deflazacort
Unknown Company
Corticosteroid [EPC]oral2025

Competitive Landscape

20 companies ranked by most advanced pipeline stage

PTC Therapeutics
14 programs
2
8
3
1
AtalurenPhase 31 trial
AtalurenPhase 31 trial
DeflazacortPhase 31 trial
AtalurenPhase 21 trial
AtalurenPhase 21 trial
+9 more programs
Active Trials
NCT02592941Approved For Marketing
NCT02295748Completed24Est. Aug 2017
NCT02251600Completed24Est. Oct 2015
+11 more trials
Pfizer
PfizerNEW YORK, NY
4 programs
1
1
fordadistrogene movaparvovecPhase 3Gene Therapy1 trial
PF-06939926Phase 11 trial
Visit frequencyN/A1 trial
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of FamiliesN/A1 trial
Active Trials
NCT03760029Completed312Est. Mar 2023
NCT03680365Completed60Est. Mar 2019
NCT03362502Terminated23Est. Jul 2025
+1 more trials
Roche
RocheSTAVANGER NORWAY, Norway
3 programs
1
1
delandistrogene moxeparvovecPhase 3Gene Therapy3 trials
SatralizumabPhase 2Monoclonal Antibody1 trial
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of FamiliesN/A
Active Trials
NCT06450639Recruiting50Est. Jun 2028
NCT06128564Active Not Recruiting13Est. Jan 2034
NCT05967351Enrolling By Invitation400Est. Oct 2033
+1 more trials
BioMarin Pharmaceutical
7 programs
4
2
BMN 044 IV 6 mg/kgPhase 21 trial
PRO044 SC 6 mg/kgPhase 21 trial
BMN 351Phase 1/21 trial
PRO044 SCPhase 1/21 trial
PRO045, 0.15 mg/kg/weekPhase 1/21 trial
+2 more programs
Active Trials
NCT02636686No Longer Available
NCT06280209Recruiting18Est. Sep 2026
NCT01037309Completed18Est. Oct 2013
+4 more trials
Capricor Therapeutics
4 programs
3
Allogeneic Cardiosphere-Derived CellsPhase 21 trial
Allogeneic Cardiosphere-Derived CellsPhase 21 trial
DeramiocelPhase 2Cell Therapy1 trial
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of FamiliesN/A
Active Trials
NCT02485938Completed25Est. Sep 2017
NCT06304064Completed8Est. Mar 2019
NCT04428476Active Not Recruiting13Est. May 2026
Satellos Bioscience
Satellos BioscienceTORONTO, Ontario, Canada
2 programs
1
1
SAT-3247Phase 21 trial
SAT-3247Phase 11 trial
Active Trials
NCT06565208Completed77Est. Apr 2025
NCT07287189Recruiting51Est. Jun 2027
DS
Daiichi SankyoChina - Shanghai
2 programs
1
1
DS-5141bPhase 21 trial
DS-5141bPhase 1/21 trial
Active Trials
NCT02667483Completed8Est. Oct 2020
NCT04433234Active Not Recruiting8Est. Jan 2027
Processa Pharmaceuticals
2 programs
1
1
HT-100Phase 21 trial
HT-100Phase 1/21 trial
Active Trials
NCT01847573Terminated17Est. Mar 2016
NCT01978366Terminated17Est. Apr 2016
Sanofi
SanofiPARIS, France
2 programs
2
AtalurenPhase 2
AtalurenPhase 2
Edgewise Therapeutics
2 programs
2
Sevasemten Dose 1Phase 21 trial
Sevasemten Dose 1Phase 21 trial
Active Trials
NCT06100887Active Not Recruiting43Est. Mar 2027
NCT05540860Active Not Recruiting76Est. Jan 2027
M&
Merck & Co.RAHWAY, NJ
1 program
1
ACE-031Phase 21 trial
Active Trials
NCT01239758Terminated11Est. May 2011
United Therapeutics
United TherapeuticsMD - Silver Spring
1 program
1
AVI-4658Phase 1/21 trial
Active Trials
NCT00159250Completed7Est. Mar 2009
Ipsen
IpsenChina - Tianjin
1 program
1
IGF-1Phase 1/21 trial
Active Trials
NCT01207908Completed44Est. Jun 2013
Alliance Pharmaceuticals
1 program
1
rAAV1.CMV.huFollistin344Phase 1/21 trial
Active Trials
NCT02354781Completed3Est. Nov 2017
Astellas
AstellasChina - Shenyang
1 program
1
scAAV9.U7.ACCAPhase 1/21 trial
Active Trials
NCT04240314Completed3Est. Jul 2025
Insmed
InsmedBRIDGEWATER, NJ
1 program
1
INS1201Phase 11 trial
Active Trials
NCT06817382Recruiting12Est. Mar 2028
Hansa Biopharma
Hansa BiopharmaSweden - Lund
1 program
1
delandistrogene moxeparvovecPhase 1Gene Therapy1 trial
Active Trials
NCT06241950TerminatedEst. Oct 2025
AskBio
AskBioNC - Durham
1 program
1
rAAV2.5-CMV-minidystrophinPhase 11 trial
Active Trials
NCT00428935Completed6Est. Jul 2010
RespirTech
RespirTechMN - Plymouth
1 program
Peak Cough Flow and Cough Clearance in Patients With Muscular DystrophyN/A1 trial
Active Trials
NCT02034305CompletedEst. Jan 2021
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
1 program
VamoroloneN/A1 trial
Active Trials
NCT06564974RecruitingEst. Feb 2032

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Rochedelandistrogene moxeparvovec
Pfizerfordadistrogene movaparvovec
PTC TherapeuticsDeflazacort
PTC TherapeuticsAtaluren
PTC TherapeuticsAtaluren
Satellos BioscienceSAT-3247
RocheSatralizumab
Edgewise TherapeuticsSevasemten Dose 1
Rochedelandistrogene moxeparvovec
Edgewise TherapeuticsSevasemten Dose 1
Capricor TherapeuticsDeramiocel
Daiichi SankyoDS-5141b
PTC TherapeuticsAtaluren
PTC TherapeuticsAtaluren
Capricor TherapeuticsAllogeneic Cardiosphere-Derived Cells

Showing 15 of 48 trials with date data

Clinical Trials (50)

Total enrollment: 2,338 patients across 50 trials

NCT05967351Rochedelandistrogene moxeparvovec

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

Start: Sep 2023Est. completion: Oct 2033400 patients
Phase 3Enrolling By Invitation
NCT05689164Pfizerfordadistrogene movaparvovec

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

Start: Mar 2023Est. completion: Sep 20257 patients
Phase 3Terminated
NCT03642145PTC TherapeuticsDeflazacort

A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

Start: Oct 2018Est. completion: Jul 20210
Phase 3Withdrawn
NCT01557400PTC TherapeuticsAtaluren

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Start: May 2012Est. completion: Jan 201894 patients
Phase 3Completed
NCT01247207PTC TherapeuticsAtaluren

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

Start: Nov 2010Est. completion: Feb 2026270 patients
Phase 3Completed
NCT07287189Satellos BioscienceSAT-3247

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Start: Dec 2025Est. completion: Jun 202751 patients
Phase 2Recruiting
NCT06450639RocheSatralizumab

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

Start: Apr 2025Est. completion: Jun 202850 patients
Phase 2Recruiting
NCT06100887Edgewise TherapeuticsSevasemten Dose 1

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

Start: Mar 2024Est. completion: Mar 202743 patients
Phase 2Active Not Recruiting
NCT06128564Rochedelandistrogene moxeparvovec

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)

Start: Nov 2023Est. completion: Jan 203413 patients
Phase 2Active Not Recruiting
NCT05540860Edgewise TherapeuticsSevasemten Dose 1

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

Start: Oct 2022Est. completion: Jan 202776 patients
Phase 2Active Not Recruiting
NCT04428476Capricor TherapeuticsDeramiocel

Open-label Extension of the HOPE-2 Trial

Start: Aug 2020Est. completion: May 202613 patients
Phase 2Active Not Recruiting
NCT04433234Daiichi SankyoDS-5141b

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Start: Jun 2020Est. completion: Jan 20278 patients
Phase 2Active Not Recruiting
NCT03796637PTC TherapeuticsAtaluren

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Start: Apr 2019Est. completion: Jun 20196 patients
Phase 2Completed
NCT03648827PTC TherapeuticsAtaluren

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Start: Dec 2018Est. completion: Oct 202020 patients
Phase 2Completed
NCT06304064Capricor TherapeuticsAllogeneic Cardiosphere-Derived Cells

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

Start: Jun 2018Est. completion: Mar 20198 patients
Phase 2Completed
NCT02819557PTC TherapeuticsAtaluren

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

Start: Jun 2016Est. completion: Feb 201814 patients
Phase 2Completed
NCT02958202BioMarin PharmaceuticalBMN 044 IV 6 mg/kg

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

Start: Apr 2016Est. completion: Sep 20167 patients
Phase 2Terminated
NCT02485938Capricor TherapeuticsAllogeneic Cardiosphere-Derived Cells

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

Start: Jan 2016Est. completion: Sep 201725 patients
Phase 2Completed
NCT02329769BioMarin PharmaceuticalPRO044 SC 6 mg/kg

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Start: Dec 2014Est. completion: Aug 201615 patients
Phase 2Terminated
NCT01978366Processa PharmaceuticalsHT-100

Open Label Extension Study of HT-100 in Patients With DMD

Start: Oct 2013Est. completion: Apr 201617 patients
Phase 2Terminated
NCT01239758Merck & Co.ACE-031

Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

Start: Oct 2010Est. completion: May 201111 patients
Phase 2Terminated
NCT01009294PTC TherapeuticsAtaluren

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

Start: Jan 2010Est. completion: Mar 20106 patients
Phase 2Terminated
NCT00847379PTC TherapeuticsAtaluren

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Start: Jan 2009Est. completion: May 2010173 patients
Phase 2Terminated
NCT00759876PTC TherapeuticsAtaluren

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

Start: Aug 2008Est. completion: May 201036 patients
Phase 2Terminated
NCT00592553PTC TherapeuticsAtaluren

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Start: Feb 2008Est. completion: Dec 2009174 patients
Phase 2Completed
NCT00264888PTC TherapeuticsPTC124

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

Start: Dec 2005Est. completion: May 200738 patients
Phase 2Completed
NCT06280209BioMarin PharmaceuticalBMN 351

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Start: Jan 2024Est. completion: Sep 202618 patients
Phase 1/2Recruiting
NCT04240314AstellasscAAV9.U7.ACCA

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

Start: Jan 2020Est. completion: Jul 20253 patients
Phase 1/2Completed
NCT02667483Daiichi SankyoDS-5141b

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Start: Oct 2015Est. completion: Oct 20208 patients
Phase 1/2Completed
NCT02354781Alliance PharmaceuticalsrAAV1.CMV.huFollistin344

Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

Start: Jan 2015Est. completion: Nov 20173 patients
Phase 1/2Completed
NCT01957059BioMarin PharmaceuticalRegimen Selection Phase Group 2

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

Start: Jun 2013Est. completion: Aug 20169 patients
Phase 1/2Terminated
NCT01847573Processa PharmaceuticalsHT-100

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

Start: May 2013Est. completion: Mar 201617 patients
Phase 1/2Terminated
NCT01826474BioMarin PharmaceuticalPRO045, 0.15 mg/kg/week

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

Start: Jan 2013Est. completion: Aug 201615 patients
Phase 1/2Terminated
NCT01207908IpsenIGF-1

Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

Start: Nov 2010Est. completion: Jun 201344 patients
Phase 1/2Completed
NCT01037309BioMarin PharmaceuticalPRO044 SC

Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Start: Dec 2009Est. completion: Oct 201318 patients
Phase 1/2Completed
NCT00159250United TherapeuticsAVI-4658

Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

Start: Oct 2007Est. completion: Mar 20097 patients
Phase 1/2Completed
NCT06817382InsmedINS1201

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Start: Jul 2025Est. completion: Mar 202812 patients
Phase 1Recruiting
NCT06565208Satellos BioscienceSAT-3247

First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort

Start: Aug 2024Est. completion: Apr 202577 patients
Phase 1Completed
NCT06241950Hansa Biopharmadelandistrogene moxeparvovec

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)

Start: Jan 2024Est. completion: Oct 2025
Phase 1Terminated
NCT04626674Rochedelandistrogene moxeparvovec

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Start: Nov 2020Est. completion: Feb 202883 patients
Phase 1Recruiting
NCT03362502PfizerPF-06939926

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

Start: Jan 2018Est. completion: Jul 202523 patients
Phase 1Terminated
NCT02251600PTC TherapeuticsDeflazacort

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Start: Dec 2014Est. completion: Oct 201524 patients
Phase 1Completed
NCT02295748PTC TherapeuticsDeflazacort

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

Start: Dec 2014Est. completion: Aug 201724 patients
Phase 1Completed
NCT00428935AskBiorAAV2.5-CMV-minidystrophin

Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

Start: Mar 2006Est. completion: Jul 20106 patients
Phase 1Completed
NCT02592941PTC TherapeuticsDeflazacort

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

N/AApproved For Marketing
NCT02636686BioMarin PharmaceuticalDrisapersen

Extension Study of Drisapersen in DMD Subjects

N/ANo Longer Available
NCT06564974Catalyst PharmaceuticalsVamorolone

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Start: Sep 2024Est. completion: Feb 2032
N/ARecruiting
NCT03760029PfizerVisit frequency

A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

Start: Jul 2019Est. completion: Mar 2023312 patients
N/ACompleted
NCT03680365PfizerYour Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

Start: Sep 2018Est. completion: Mar 201960 patients
N/ACompleted
NCT02034305RespirTechPeak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

Start: Jan 2014Est. completion: Jan 2021
N/ACompleted

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

5 late-stage (Phase 3) programs — potential near-term approvals
6 actively recruiting trials targeting 2,338 patients
20 companies competing in this space

Related Indications

Cystic Fibrosis(244)Hemophilia A(139)Sickle Cell Disease(64)Fabry Disease(55)Hemophilia B(47)Spinal Muscular Atrophy(34)Phenylketonuria(32)Gaucher Disease(29)Severe Hemophilia A(26)Pompe Disease(20)