Condition (MeSH)
Myotonic Dystrophy
ClinicalTrials.gov lists 124 trials for Myotonic Dystrophy or a narrower condition beneath it. Lupin Ltd. has the most among industry and government lead sponsors, with 6.
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Trials per phase (log scale)
124 of the 124 trials are tagged with Myotonic Dystrophy itself; the rest are tagged with a narrower condition beneath it.
When did Myotonic Dystrophy trials start? (24)
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
- Not applicable
- Observational
Top of scale: 13
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.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 8 |
| 2025 | 13 |
| 2024 | 13 |
| 2023 | 9 |
| 2022 | 9 |
| 2021 | 13 |
| 2020 | 8 |
| 2019 | 4 |
| 2018 | 2 |
| 2017 | 3 |
Showing 10 of 24 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 10 trials starting in 2026 and about 13 in 2027, the average of the 24 months through September, counted as registered and carried forward. 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 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. 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 20 trials still running at the 2026-10-09 snapshot; registered median of 26 months across 58 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Myotonic Dystrophy trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 5 |
| Phase 1/2 | 8 |
| Phase 2 | 8 |
| Phase 2/3 | 4 |
| Phase 3 | 13 |
| Not applicable | 30 |
| Observational | 55 |
| Expanded access | 1 |
How many Myotonic Dystrophy trials are recruiting? (9)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 3 |
| Recruiting | 34 |
| Enrolling by invitation | 10 |
| Active, not recruiting | 6 |
| Available | 1 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Myotonic Dystrophy trials? (5)
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.
| Sponsor | Trials |
|---|---|
| Lupin Ltd. | 6 |
| Avidity Biosciences, Inc. | 5 |
| AMO Pharma Limited | 4 |
| Dyne Therapeutics | 3 |
| PepGen Inc | 3 |
Which drugs are being tested for Myotonic Dystrophy? (3)
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.
| Drug | Trials |
|---|---|
| Mexiletine | 4 |
| Tideglusib | 4 |
| DYNE-101 | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| AOC 1001 | 3 |
| PGN-EDODM1 | 2 |
| VX-670 | 2 |
| Exercise | 2 |
| Mexiletine granules for prolonged-release oral suspension | 2 |
Showing 5 of 12 interventions.
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 Myotonic Dystrophy trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Fall | 4 | 1 |
| Respiratory Failure | 4 | 2 |
| Acute Inflammation of Gallbladder | 1 | 1 |
| Bundle Branch Block Left | 1 | 1 |
| Chest Pain | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Myotonic Dystrophy? (134)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| The Splice Index as a prognostic biomarker of strength and function in myotonic dystrophy type 1. | The Journal of clinical investigation | 2025 | Yes |
| Efficacy of Manual Wheelchair Skills Training for Improving Skills and Confidence in People With Hereditary Degenerative Disorders: Protocol for a Sequential Multimethods Study. | JMIR research protocols | 2025 | Yes |
| Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study. | PloS one | 2025 | Yes |
| Neurobehavioral Phenotype of Children With Congenital Myotonic Dystrophy. | Neurology | 2024 | Yes |
| Clinical features and genetic spectrum of a multicenter Chinese cohort with myotonic dystrophy type 1. | Orphanet journal of rare diseases | 2024 | Yes |
Showing 5 of 134 publications.
Prevalence (36)
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.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Myotonic dystrophy | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Myotonic dystrophy | Point prevalence | 1-9 / 100 000 | United States | US | Unspecified |
| Myotonic dystrophy | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Myotonic dystrophy | Point prevalence | 1-5 / 10 000 | Finland | Not stated | Exact |
| Myotonic dystrophy | Point prevalence | 1-9 / 100 000 | Japan | Not stated | Exact |
Showing 5 of 36 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Myotonic dystrophy | 206647 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Myotonic dystrophy | 206647 | 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.