The DMD trial landscape in 2026
Duchenne muscular dystrophy has become one of the most complex and commercially consequential rare disease settings in clinical development. Sarepta Therapeutics' approval of delandistrogene moxeparvovec (Elevidys), the first systemic gene therapy for DMD, marked a pivotal moment, even as its regulatory path and clinical evidence base remain subjects of ongoing debate. The gene therapy landscape is now a multi-sponsor competition, with Solid Biosciences, BioMarin (fordadistrogene movaparvovec), and Pfizer (SRP-9003) all pursuing systemic AAV micro-dystrophin programs.
Simultaneously, the exon-skipping pipeline is maturing. Eteplirsen (exon 51 skip, Sarepta) paved the regulatory way; golodirsen and viltolarsen (exon 53) followed. Casimersen (exon 45) extended this strategy further. Entrada Therapeutics is developing endosomal escape technology to improve oligonucleotide delivery, with its ENTR-701 (exon 44) and ENTR-601-44 programs representing the next generation.
Key DMD program categories to monitor:
- Systemic gene therapy (AAV micro-dystrophin): Sarepta Elevidys follow-on studies, Solid Biosciences SGT-001, BioMarin fordadistrogene, Pfizer SRP-9003
- Exon skipping (antisense oligonucleotides): Next-generation exon 44, 45, 51, 53 skippers with improved delivery
- Endosomal escape technology: Entrada Therapeutics ENTR-701 and ENTR-601-44 programs
- Utrophin upregulation: Ezutromid and next-generation small molecule upregulators
- Cardiac protection: Programs specifically targeting DMD cardiomyopathy, a major cause of morbidity in older patients
- Combination approaches: Gene therapy plus exon skipping, or gene therapy plus cardiac protection
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Get the Free DigestA primary completion date pushed out, enrollment cut, or a primary endpoint changed in the last 90 days. Active trials only.
| Trial | Warning signs | Status |
|---|---|---|
| An Open-label Long-term Follow-up Study of SAT-3247 for Participants With… NCT06867107 | Primary completion moved at least 1 year 1 month later Primary endpoint added | Enrolling by Invitation |
6 more on the Trials at Risk page. See which trials →
Phase 2 and Phase 3 trials with a primary completion date in the next 180 days, as ClinicalTrials.gov estimates it today. Active trials only.
| Trial | Scheduled | Phase |
|---|---|---|
| Microdystrophin Gene Transfer Study in Adolescents and Children With DMD NCT03368742 | Oct 15, 2026 · Estimated | Phase 1/2 |
9 more on the Completions page. See the calendar →
Who uses DMD trial monitoring
Rare disease BD professionals
DMD is one of the most commercially valuable rare disease settings. Sarepta built a multi-billion dollar franchise on exon-skipping antisense oligonucleotides. The gene therapy approvals, with price tags exceeding $3 million per patient, make DMD the highest per-patient revenue opportunity in neuromuscular disease. BD teams track pipeline entries to identify early-stage assets for in-licensing and assess competitive threats to existing franchise assets.
Gene therapy investors
Analysts tracking gene therapy use DMD trial registrations as indicators of program maturity and competitive positioning. The question of whether any follow-on micro-dystrophin program (BioMarin, Solid Biosciences) can demonstrate superiority to Elevidys on functional outcomes, and whether the accelerated approval standard will hold for the category, is the central investment thesis question in DMD gene therapy.
Patient advocacy-engaged companies
Parent Project Muscular Dystrophy and Cure Duchenne have unusually active roles in DMD clinical development. Companies developing in DMD must maintain awareness of both competitor programs and advocacy preferences, particularly around exon-specific patient subpopulations who are eligible for different treatments.
Frequently asked questions
132 active trials, 62 recruiting.
Industry sponsors run 55 of these trials. The rest are run by universities, hospitals, government bodies and other non-commercial sponsors. The table below lists the 5 largest of 31.
| Top industry sponsor | Active trials |
|---|---|
| Sarepta Therapeutics, Inc. | 7 |
| Entrada Therapeutics, Inc. | 3 |
| Italfarmaco | 3 |
| NS Pharma, Inc. | 3 |
| REGENXBIO Inc. | 3 |
View the full Duchenne Muscular Dystrophy pipeline on the dashboard
| Trial | Status | Latest tracked change | Seen |
|---|---|---|---|
| Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of… NCT07356063 | Active, Not Recruiting | Interventions modified | 2026-10-10 |
| Evaluating VM100 Nutritional Supplement for Improving Quality of Life in… NCT07766980 | Recruiting | Interventions modified | 2026-10-09 |
| Registry for Duchenne and Becker Muscular Dystrophy NCT07402122 | Recruiting | Trial sites expanded: 2 → 30 locations | 2026-10-09 |
| Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With… NCT07542314 | Recruiting | Recruitment opened | 2026-10-07 |
| Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular… NCT04433234 | Active, Not Recruiting | Completion pushed from Jan 31, 2027 to Jan 31, 2031 | 2026-10-07 |
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