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Condition (MeSH)

Muscular Dystrophy, Duchenne

ClinicalTrials.gov lists 476 trials for Muscular Dystrophy, Duchenne or a narrower condition beneath it. Sarepta Therapeutics, Inc. has the most among industry and government lead sponsors, with 27.

MeSH D020388 Musculoskeletal Diseases C05 C10 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 57 Phase 1/2 Phase 1/2 45 Phase 2 Phase 2 84 Phase 2/3 Phase 2/3 10 Phase 3 Phase 3 49 Phase 4 Phase 4 10 Not applicable Not applicable 85 Observational Observational 130 Phase 1 Phase 1 57 Phase 1/2 Phase 1/2 45 Phase 2 Phase 2 84 Phase 2/3 Phase 2/3 10 Phase 3 Phase 3 49 Phase 4 Phase 4 10 Not applicable Not applicable 85 Observational Observational 130

Trials per phase

1 more section with rows

476 of the 476 trials are tagged with Muscular Dystrophy, Duchenne itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Muscular Dystrophy, Duchenne trials start? (27)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 43

1995: 1 trials (Phase 3 1) 2000: 2 trials (Phase 2/3 1, Phase 1 1) 2001: 1 trials (Phase 2 1) 2002: 3 trials (Observational 1, Phase 2 1, Phase 1/2 1) 2004: 2 trials (Observational 1, Phase 3 1) 2005: 5 trials (Observational 1, Phase 2 2, Phase 1/2 2) 2006: 5 trials (Observational 1, Not applicable 2, Phase 2 1, Phase 1 1) 2007: 6 trials (Observational 2, Phase 4 1, Phase 3 1, Phase 1/2 1, Phase 1 1) 2008: 6 trials (Phase 4 1, Phase 3 1, Phase 2 3, Phase 1 1) 2009: 9 trials (Observational 1, Not applicable 1, Phase 3 2, Phase 2 1, Phase 1/2 2, Phase 1 2) 2010: 18 trials (Observational 5, Phase 4 1, Phase 3 3, Phase 2/3 2, Phase 2 5, Phase 1/2 1, Phase 1 1) 2011: 15 trials (Observational 4, Not applicable 2, Phase 4 1, Phase 3 1, Phase 2 3, Phase 1/2 1, Phase 1 3) 2012: 17 trials (Observational 8, Not applicable 2, Phase 3 2, Phase 2 1, Phase 1 4) 2013: 30 trials (Observational 6, Not applicable 5, Phase 4 1, Phase 3 5, Phase 2/3 1, Phase 2 3, Phase 1/2 6, Phase 1 3) 2014: 22 trials (Observational 4, Not applicable 3, Phase 3 3, Phase 2 5, Phase 1/2 3, Phase 1 4) 2015: 21 trials (Observational 5, Not applicable 4, Phase 3 1, Phase 2 2, Phase 1/2 4, Phase 1 5) 2016: 23 trials (Observational 2, Not applicable 3, Phase 3 2, Phase 2 11, Phase 1/2 3, Phase 1 2) 2017: 20 trials (Observational 6, Not applicable 4, Phase 3 2, Phase 2/3 2, Phase 2 3, Phase 1/2 2, Phase 1 1) 2018: 34 trials (Observational 8, Not applicable 7, Phase 3 5, Phase 2 7, Phase 1/2 3, Phase 1 4) 2019: 22 trials (Observational 9, Not applicable 3, Phase 3 1, Phase 2/3 2, Phase 2 4, Phase 1/2 2, Phase 1 1) 2020: 22 trials (Observational 5, Not applicable 3, Phase 4 1, Phase 3 5, Phase 2 4, Phase 1/2 1, Phase 1 3) 2021: 29 trials (Observational 8, Not applicable 8, Phase 4 2, Phase 3 4, Phase 2/3 1, Phase 2 2, Phase 1/2 1, Phase 1 3) 2022: 29 trials (Observational 14, Not applicable 5, Phase 3 1, Phase 2 5, Phase 1/2 2, Phase 1 2) 2023: 23 trials (Observational 6, Not applicable 9, Phase 3 3, Phase 2/3 1, Phase 2 3, Phase 1/2 1) 2024: 43 trials (Observational 16, Not applicable 6, Phase 4 1, Phase 3 1, Phase 2 7, Phase 1/2 4, Phase 1 8) 2025: 29 trials (Observational 7, Not applicable 8, Phase 3 1, Phase 2 5, Phase 1/2 3, Phase 1 5)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 31
2025 29
2024 43
2023 23
2022 29
2021 29
2020 22
2019 22
2018 34
2017 20

Showing 10 of 27 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 39 trials starting in 2026 and about 35 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 56 trials still running at the 2026-10-09 snapshot; registered median of 24 months across 288 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Muscular Dystrophy, Duchenne trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 57
Phase 1/2 45
Phase 2 84
Phase 2/3 10
Phase 3 49
Phase 4 10
Not applicable 85
Observational 130
Expanded access 6

How many Muscular Dystrophy, Duchenne trials are recruiting? (11)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 19
Recruiting 58
Enrolling by invitation 18
Active, not recruiting 34
Available 2

Showing 5 of 11 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Muscular Dystrophy, Duchenne trials? (11)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Sarepta Therapeutics, Inc. 27
PTC Therapeutics 18
Cooperative International Neuromuscular Research Group 15
BioMarin Pharmaceutical 10
NS Pharma, Inc. 9

Showing 5 of 11 sponsors.

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Which drugs are being tested for Muscular Dystrophy, Duchenne? (12)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Ataluren 13
Eteplirsen 11
Viltolarsen 8
Prednisone 8
Givinostat 6

Showing 5 of 12 drugs.

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Interventions as named in the registry (3)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Tadalafil Pill 6
Vamorolone 5
(+)- Epicatechin 4

These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Muscular Dystrophy, Duchenne trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Femur fracture 95 23
Pneumonia 40 14
Rhabdomyolysis 25 15
Vomiting 22 13
Influenza 18 11

Showing 5 of 10 adverse events.

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Which publications cover Muscular Dystrophy, Duchenne? (472)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
The Potential of Targeting APE1/Ref-1 as a Therapeutic Intervention for Duchenne Muscular Dystrophy. Antioxidants & redox signaling 2025 Yes
Effect of Tadalafil on cardiac function and left ventricular dimensions in Duchenne muscular dystrophy: safety and cardiac MRI substudy results from a randomized, placebo-controlled trial. BMC cardiovascular disorders 2025 Yes
Mineralocorticoid receptor antagonism of vamorolone: Evidence from LIONHEART and VISION-DMD clinical trials. Steroids 2025 Yes
A model-informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy. CPT: pharmacometrics & systems pharmacology 2025 Yes
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nature medicine 2025 Yes

Showing 5 of 472 publications.

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Prevalence (45)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Duchenne muscular dystrophy Prevalence at birth 1-9 / 100 000 United Kingdom GB Exact
Duchenne muscular dystrophy Prevalence at birth 1-9 / 100 000 United Kingdom GB Unspecified
Duchenne muscular dystrophy Point prevalence 1-9 / 100 000 United Kingdom GB Exact
Duchenne muscular dystrophy Point prevalence 1-9 / 100 000 United Kingdom GB Unspecified
Duchenne muscular dystrophy Prevalence at birth 1-9 / 100 000 United Kingdom GB Exact

Showing 5 of 45 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Duchenne muscular dystrophy 98896 Exact Orphanet Rare Disease Ontology (ORDO)
Duchenne muscular dystrophy 98896 Exact Orphadata Science knowledge files, Product 1

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

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