Muscular Dystrophy: cell-therapy evidence by therapy class
Across every stem-cell intervention, ClinicalTrials.gov holds 26 registered records for Muscular Dystrophy — 4 recruiting, 0 at Phase 3 or 4, in 17 countries. Snapshot 2026-08-29.
The condition, and why cells are proposed
The muscular dystrophies are genetic disorders of muscle structural proteins; Duchenne is the most studied. Cell approaches face a fundamental delivery problem — skeletal muscle is distributed throughout the body — and gene-targeted therapies have moved faster than cell replacement for exactly that reason.
Therapy classes side by side
| Therapy class | Registered | Recruiting | Phase 3/4 | Countries |
|---|---|---|---|---|
| MSC | 9 | 0 | 0 | 5 |
| HSCT | 1 | 1 | 0 | 1 |
| exosomes | 1 | 0 | 0 | 1 |
| PRP | 0 | 0 | 0 | 0 |
Counts come from separate registry queries per class; a study testing two interventions can appear in more than one row, so rows do not sum to the all-interventions total.
Geography of the research
United States accounts for about 35% of country mentions among sampled studies; 17 countries host at least one site.
| Country | Studies with a site there |
|---|---|
| United States | 9 |
| India | 6 |
| Turkey (Türkiye) | 2 |
| South Korea | 2 |
| Australia | 2 |
| Jordan | 1 |
| Israel | 1 |
| Iran | 1 |
The largest registered studies
Sorted by planned enrolment — size is not quality, but a 300-participant trial can answer questions a 12-participant series cannot.
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT00874783 — Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases | n/a | Recruiting | 120 | Hadassah Medical Organization | 2009-04 |
| NCT02413450 — Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias | n/a | Enrolling By Invitation | 100 | Johns Hopkins University | 2013-08 |
| NCT06328725 — Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy | Phase 1, Phase 2 | Unknown | 88 | ENCell | 2024-03 |
| NCT06565208 — First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort | Early Phase 1 | Completed | 77 | Satellos Bioscience, Inc. | 2024-08-21 |
| NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients | Phase 2 | Recruiting | 51 | Satellos Bioscience, Inc. | 2025-12-08 |
| NCT06579352 — Study of UC-MSC in DMD Patients | Phase 2 | Active Not Recruiting | 35 | MED Institute Inc. | 2024-08-05 |
Most recently registered
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients | n/a | Recruiting | n/a | Satellos Bioscience, Inc. | 2025-12-08 |
| NCT07086521 — Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) | n/a | Recruiting | n/a | Restem, LLC. | 2025-12-01 |
| NCT06692426 — Trial of Cell Based Therapy for DMD | n/a | Recruiting | n/a | Masonic Cancer Center, University of Minnesota | 2025-03-20 |
| NCT06565208 — First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort | n/a | Completed | n/a | Satellos Bioscience, Inc. | 2024-08-21 |
| NCT06579352 — Study of UC-MSC in DMD Patients | n/a | Active Not Recruiting | n/a | MED Institute Inc. | 2024-08-05 |
Reading this honestly
These figures describe effort, not outcome. Published results, with their control arms and endpoints, are where benefit is decided. For Muscular Dystrophy specifically, the practical question is which registered study — if any — matches the product a provider is offering, and what its outcome measures actually are.
Frequently asked
How many stem-cell trials exist for Muscular Dystrophy?
26 registered records mention a stem-cell intervention for this condition (2026-08-29 snapshot), of which 4 were recruiting.
Which therapy class is furthest along for Muscular Dystrophy?
Ranked by Phase 3/4 records: MSC (0), HSCT (0), exosomes (0), PRP (0).
Does a recruiting trial mean I can be treated?
No. Trials have eligibility criteria, and joining one is not the same as buying a treatment. Paying a clinic for an unproven intervention outside a registered study gives you neither the protections nor the data collection of a trial.
Registry and literature counts retrieved 2026-08-29 (ClinicalTrials.gov API v2; PubMed E-utilities). Registration or publication volume measures research activity, not effectiveness or approval. Educational information — not medical advice; verify product, indication, legal pathway and evidence with an independent qualified physician. Page generated 2026-08-29. See our editorial standards.
Download the query data
Query totals are full API matches; status, phase and country counts describe the first up to 400 returned records per query. Query classes overlap and must not be summed as unique trials. Counts are not treatment outcomes. Snapshot: 2026-08-29. Methods and field definitions.