Condition (MeSH)
Myopathies, Nemaline
ClinicalTrials.gov lists 12 trials for Myopathies, Nemaline or a narrower condition beneath it. Vastra Gotaland Region has the most among industry and government lead sponsors, with 1.
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Trials per phase
12 of the 12 trials are tagged with Myopathies, Nemaline itself; the rest are tagged with a narrower condition beneath it.
When did Myopathies, Nemaline trials start? (7)
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 2
- Not applicable
- Observational
Top of scale: 2
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) | 3 |
| 2025 | 2 |
| 2024 | 2 |
| 2021 | 1 |
| 2018 | 2 |
| 2014 | 1 |
| 2003 | 1 |
No forecast is made for this condition: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Myopathies, Nemaline trials in? (3)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 2 | 1 |
| Not applicable | 4 |
| Observational | 7 |
How many Myopathies, Nemaline trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 3 |
| Recruiting | 5 |
| Active, not recruiting | 1 |
| Completed | 2 |
| Unknown status | 1 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Myopathies, Nemaline 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 |
|---|---|
| Vastra Gotaland Region | 1 |
Which drugs are being tested for Myopathies, Nemaline? (2)
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 |
|---|---|
| Tyrosine | 1 |
| Albuterol | 1 |
Interventions as named in the registry (2)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| QOL assessment, Neuropsychological | 1 |
| Salbutamol Only Product in Oral Dose Form | 1 |
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 publications cover Myopathies, Nemaline? (16)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Changes in cross-bridge cycling underlie muscle weakness in patients with tropomyosin 3-based myopathy. | Human molecular genetics | 2011 | Yes |
| Altered myofilament function depresses force generation in patients with nebulin-based nemaline myopathy (NEM2). | Journal of structural biology | 2010 | Yes |
| Mutations of tropomyosin 3 (TPM3) are common and associated with type 1 myofiber hypotrophy in congenital fiber type disproportion. | Human mutation | 2010 | Yes |
| The exon 55 deletion in the nebulin gene--one single founder mutation with world-wide occurrence. | Neuromuscular disorders : NMD | 2009 | Yes |
| Thin filament length dysregulation contributes to muscle weakness in nemaline myopathy patients with nebulin deficiency. | Human molecular genetics | 2009 | Yes |
Showing 5 of 16 publications.
Prevalence (6)
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 |
|---|---|---|---|---|---|
| Nemaline myopathy | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
| Nemaline myopathy | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Nemaline myopathy | Point prevalence | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Nemaline myopathy | Prevalence at birth | 1-9 / 100 000 | Europe | Not stated | Exact |
| Nemaline myopathy | Prevalence at birth | 1-9 / 100 000 | Europe | Not stated | Exact |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Nemaline myopathy | 607 | Exact | Orphadata Science knowledge files, Product 1 |
| Nemaline myopathy | 607 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.