Muscular Dystrophy, Duchenne

Rare Diseases
4
Pipeline Programs
3
Companies
5
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
3
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

PTC Therapeutics
4 programs
3
AtalurenPhase 31 trial
AtalurenPhase 31 trial
AtalurenPhase 31 trial
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)N/A1 trial
Active Trials
NCT02369731Completed316Est. Mar 2025
NCT03179631Completed360Est. Jul 2023
NCT01826487Completed230Est. Aug 2015
+1 more trials
Pfizer
PfizerNEW YORK, NY
1 program
1
PF-06939926Phase 21 trial
Active Trials
NCT05429372Terminated10Est. Oct 2025
NMD Pharma
NMD PharmaDenmark - Aarhus
1 program
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)N/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
PTC TherapeuticsAtaluren
PTC TherapeuticsAtaluren
PTC TherapeuticsAtaluren
PfizerPF-06939926
PTC TherapeuticsRegistry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Clinical Trials (5)

Total enrollment: 1,135 patients across 5 trials

NCT03179631PTC TherapeuticsAtaluren

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Start: Jul 2017Est. completion: Jul 2023360 patients
Phase 3Completed
NCT02090959PTC TherapeuticsAtaluren

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Start: Mar 2014Est. completion: Jun 2018219 patients
Phase 3Terminated
NCT01826487PTC TherapeuticsAtaluren

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Start: Mar 2013Est. completion: Aug 2015230 patients
Phase 3Completed
NCT05429372PfizerPF-06939926

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Start: Aug 2022Est. completion: Oct 202510 patients
Phase 2Terminated
NCT02369731PTC TherapeuticsRegistry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Start: Apr 2015Est. completion: Mar 2025316 patients
N/ACompleted

Related Jobs in Rare Diseases

Manager, Gene Therapy Operations (GTO)

Genetix Biotherapeutics
8h ago
From $130K/yr

Manager, Gene Therapy Operations (GTO)

Genetix Biotherapeutics
Somerville, Massachusetts, United States
8h ago

Medical Engagement Manager – Diamyd Medical

Bonesupport
Stockholm, Sweden
Yesterday

Exec. Director, Global Patient Engagement

BioCryst Pharmaceuticals
US_North Carolina_Headquarters
Yesterday

Sr. Director, Regulatory CMC

Genetix Biotherapeutics
Somerville, MA
4d ago
From $270K/yr

Associate Director, Quality Systems

Genetix Biotherapeutics
Somerville, Massachusetts
4d ago
From $165K/yr

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

3 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space

Related Indications

Cystic Fibrosis(244)Hemophilia A(139)Sickle Cell Disease(64)Fabry Disease(55)Duchenne Muscular Dystrophy(53)Hemophilia B(47)Spinal Muscular Atrophy(34)Phenylketonuria(32)Gaucher Disease(29)Severe Hemophilia A(26)