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Condition (MeSH)

Muscular Dystrophies

ClinicalTrials.gov lists 807 trials for Muscular Dystrophies or a narrower condition beneath it. Sarepta Therapeutics, Inc. has the most among industry and government lead sponsors, with 38.

MeSH D009136 Musculoskeletal Diseases C05 C10 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 77 Phase 1/2 Phase 1/2 67 Phase 2 Phase 2 107 Phase 2/3 Phase 2/3 16 Phase 3 Phase 3 67 Phase 4 Phase 4 11 Not applicable Not applicable 180 Observational Observational 274 Phase 1 Phase 1 77 Phase 1/2 Phase 1/2 67 Phase 2 Phase 2 107 Phase 2/3 Phase 2/3 16 Phase 3 Phase 3 67 Phase 4 Phase 4 11 Not applicable Not applicable 180 Observational Observational 274

Trials per phase (log scale)

1 more section with rows

222 of the 807 trials are tagged with Muscular Dystrophies 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 Dystrophies trials start? (32)

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: 71

1993: 2 trials (Observational 2) 1995: 1 trials (Phase 3 1) 1996: 2 trials (Observational 2) 1998: 1 trials (Not applicable 1) 1999: 1 trials (Observational 1) 2000: 5 trials (Observational 3, Phase 2/3 1, Phase 1 1) 2001: 3 trials (Not applicable 1, Phase 4 1, Phase 2 1) 2002: 3 trials (Observational 1, Phase 2 1, Phase 1/2 1) 2003: 2 trials (Not applicable 1, Phase 2/3 1) 2004: 5 trials (Observational 2, Phase 3 1, Phase 2/3 1, Phase 2 1) 2005: 8 trials (Observational 3, Phase 2 2, Phase 1/2 3) 2006: 7 trials (Observational 3, Not applicable 2, Phase 2 1, Phase 1 1) 2007: 9 trials (Observational 4, Phase 4 1, Phase 3 1, Phase 2 1, Phase 1/2 1, Phase 1 1) 2008: 14 trials (Observational 1, Not applicable 3, Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 4, Phase 1 3) 2009: 15 trials (Observational 3, Not applicable 2, Phase 3 3, Phase 2 1, Phase 1/2 2, Phase 1 4) 2010: 26 trials (Observational 9, Not applicable 2, Phase 4 1, Phase 3 4, Phase 2/3 2, Phase 2 5, Phase 1/2 1, Phase 1 2) 2011: 19 trials (Observational 6, Not applicable 3, Phase 4 1, Phase 3 1, Phase 2 4, Phase 1/2 1, Phase 1 3) 2012: 27 trials (Observational 17, Not applicable 3, Phase 3 2, Phase 2 1, Phase 1 4) 2013: 44 trials (Observational 14, Not applicable 9, Phase 4 1, Phase 3 6, Phase 2/3 1, Phase 2 3, Phase 1/2 6, Phase 1 4) 2014: 40 trials (Observational 12, Not applicable 9, Phase 3 3, Phase 2 6, Phase 1/2 5, Phase 1 5) 2015: 35 trials (Observational 12, Not applicable 6, Phase 3 2, Phase 2 2, Phase 1/2 8, Phase 1 5) 2016: 42 trials (Observational 11, Not applicable 8, Phase 3 2, Phase 2 13, Phase 1/2 5, Phase 1 3) 2017: 31 trials (Observational 9, Not applicable 10, Phase 3 2, Phase 2/3 2, Phase 2 4, Phase 1/2 2, Phase 1 2) 2018: 49 trials (Observational 14, Not applicable 13, Phase 3 5, Phase 2 8, Phase 1/2 4, Phase 1 5) 2019: 43 trials (Observational 16, Not applicable 11, Phase 3 2, Phase 2/3 3, Phase 2 7, Phase 1/2 2, Phase 1 2) 2020: 39 trials (Observational 14, Not applicable 9, Phase 4 1, Phase 3 5, Phase 2 6, Phase 1/2 1, Phase 1 3) 2021: 55 trials (Observational 20, Not applicable 15, Phase 4 2, Phase 3 6, Phase 2/3 3, Phase 2 4, Phase 1/2 2, Phase 1 3) 2022: 54 trials (Observational 24, Not applicable 15, Phase 3 2, Phase 2 6, Phase 1/2 3, Phase 1 4) 2023: 47 trials (Observational 16, Not applicable 16, Phase 3 3, Phase 2/3 1, Phase 2 4, Phase 1/2 4, Phase 1 3) 2024: 71 trials (Observational 28, Not applicable 12, Phase 4 1, Phase 3 5, Phase 2 9, Phase 1/2 8, Phase 1 8) 2025: 51 trials (Observational 13, Not applicable 12, Phase 3 6, Phase 2 7, Phase 1/2 5, Phase 1 8)

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) 45
2025 51
2024 71
2023 47
2022 54
2021 55
2020 39
2019 43
2018 49
2017 31

Showing 10 of 32 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 58 trials starting in 2026 and about 56 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 96 trials still running at the 2026-10-09 snapshot; registered median of 25 months across 468 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Muscular Dystrophies trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 77
Phase 1/2 67
Phase 2 107
Phase 2/3 16
Phase 3 67
Phase 4 11
Not applicable 180
Observational 274
Expanded access 8

How many Muscular Dystrophies 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 23
Recruiting 123
Enrolling by invitation 29
Active, not recruiting 60
Available 3

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 Dystrophies trials? (7)

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. 38
PTC Therapeutics 19
Cooperative International Neuromuscular Research Group 16
Avidity Biosciences, Inc. 11
BioMarin Pharmaceutical 11

Showing 5 of 7 sponsors.

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Which drugs are being tested for Muscular Dystrophies? (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
Drisapersen 7

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
Exercise 6
Tadalafil Pill 6
Vamorolone 5

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 Dystrophies 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 96 24
Pneumonia 43 17
Rhabdomyolysis 27 16
Vomiting 22 13
Influenza 18 11

Showing 5 of 10 adverse events.

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Which publications cover Muscular Dystrophies? (194)

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

Publications
Publication Journal Year Major topic
Respiratory function in LAMA2-related muscular dystrophy and SELENON-related congenital myopathy, a 1.5-year natural history study. European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society 2024 Yes
A 5-year natural history study in LAMA2-related muscular dystrophy and SELENON-related myopathy: the Extended LAST STRONG study. BMC neurology 2024 Yes
Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies. Physical therapy 2022 Yes
User-centred assistive SystEm for arm Functions in neUromuscuLar subjects (USEFUL): a randomized controlled study. Journal of neuroengineering and rehabilitation 2021 Yes
Natural history, outcome measures and trial readiness in LAMA2-related muscular dystrophy and SELENON-related myopathy in children and adults: protocol of the LAST STRONG study. BMC neurology 2021 Yes

Showing 5 of 194 publications.

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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
Muscular dystrophy 98473 Exact Orphanet Rare Disease Ontology (ORDO)
Muscular dystrophy 98473 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (9)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: prevalence.

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