Condition (MeSH)
Distal Myopathies
ClinicalTrials.gov lists 5 trials for Distal Myopathies or a narrower condition beneath it. Bristol-Myers Squibb has the most among industry and government lead sponsors, with 1.
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Trials per phase
5 of the 5 trials are tagged with Distal Myopathies itself; the rest are tagged with a narrower condition beneath it.
When did Distal Myopathies trials start? (5)
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 2
- Not applicable
- Observational
Top of scale: 1
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 |
|---|---|
| 2025 | 1 |
| 2023 | 1 |
| 2020 | 1 |
| 2014 | 1 |
| 2008 | 1 |
No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 5 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Distal Myopathies trials in? (4)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 1 |
| Not applicable | 1 |
| Observational | 2 |
How many Distal Myopathies trials are recruiting? (4)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 1 |
| Completed | 1 |
| Terminated | 1 |
| Unknown status | 2 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Distal Myopathies trials? (1)
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 |
|---|---|
| Bristol-Myers Squibb | 1 |
Which serious adverse events do Distal Myopathies trials report? (7)
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 |
|---|---|---|
| Acute kidney injury | 1 | 1 |
| Arrhythmic storm | 1 | 1 |
| Cardiac failure congestive | 1 | 1 |
| Liver injury | 1 | 1 |
| Transient ischaemic attack | 1 | 1 |
Showing 5 of 7 adverse events.
Which publications cover Distal Myopathies? (13)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Miyoshi myopathy associated with spine rigidity and multiple contractures: a case report. | BMC musculoskeletal disorders | 2024 | Yes |
| Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan. | Orphanet journal of rare diseases | 2023 | Yes |
| Skeletal Muscle Magnetic Resonance Biomarkers in GNE Myopathy. | Neurology | 2021 | Yes |
| Population Pharmacokinetic Model of N-acetylmannosamine (ManNAc) and N-acetylneuraminic acid (Neu5Ac) in Subjects with GNE Myopathy. | Drugs in R&D | 2021 | Yes |
| Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study. | Genetics in medicine : official journal of the American College of Medical Genetics | 2021 | Yes |
Showing 5 of 13 publications.
Prevalence (2)
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 |
|---|---|---|---|---|---|
| Distal myopathy | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Distal myopathy | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Distal myopathy | 599 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Distal myopathy | 599 | 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.
Not computed for conditions with fewer than 10 trials: drugs tested.