Condition (MeSH)
Muscular Dystrophy, Oculopharyngeal
ClinicalTrials.gov lists 16 trials for Muscular Dystrophy, Oculopharyngeal or a narrower condition beneath it. Bioblast Pharma Ltd. has the most among industry and government lead sponsors, with 3.
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Trials per phase
16 of the 16 trials are tagged with Muscular Dystrophy, Oculopharyngeal itself; the rest are tagged with a narrower condition beneath it.
When did Muscular Dystrophy, Oculopharyngeal trials start? (11)
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: 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 |
|---|---|
| 2023 | 2 |
| 2022 | 2 |
| 2019 | 2 |
| 2017 | 1 |
| 2016 | 2 |
| 2015 | 1 |
| 2014 | 2 |
| 2010 | 1 |
| 2009 | 1 |
| 2004 | 1 |
Showing 10 of 11 earlier years.
No forecast is made for this condition: it averages 0.8 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 Muscular Dystrophy, Oculopharyngeal trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 1 |
| Phase 2 | 3 |
| Phase 2/3 | 1 |
| Phase 3 | 1 |
| Not applicable | 2 |
| Observational | 7 |
How many Muscular Dystrophy, Oculopharyngeal trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 4 |
| Completed | 5 |
| Terminated | 1 |
| Withdrawn | 2 |
| Unknown status | 4 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Muscular Dystrophy, Oculopharyngeal 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 |
|---|---|
| Bioblast Pharma Ltd. | 3 |
| Hillel Yaffe Medical Center | 2 |
| Sheba Medical Center | 2 |
| Benitec Biopharma, Inc. | 1 |
| National Heart, Lung, and Blood Institute (NHLBI) | 1 |
Which drugs are being tested for Muscular Dystrophy, Oculopharyngeal? (1)
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 |
|---|---|
| Trehalose | 2 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Myoblast autologous graft | 1 |
| Exercise | 1 |
| Tehalose 30gr | 1 |
| Non interventional study | 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 serious adverse events do Muscular Dystrophy, Oculopharyngeal trials report? (3)
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 |
|---|---|---|
| Aspiration | 1 | 1 |
| Pneumonia Aspiration | 1 | 1 |
| Urinary Tract Infection | 1 | 1 |
Which publications cover Muscular Dystrophy, Oculopharyngeal? (10)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| The natural history of infantile mitochondrial DNA depletion syndrome due to RRM2B deficiency. | Genetics in medicine : official journal of the American College of Medical Genetics | 2020 | Yes |
| Dysphagia-related quality of life in oculopharyngeal muscular dystrophy: Psychometric properties of the SWAL-QOL instrument. | Muscle & nerve | 2017 | Yes |
| Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study. | Molecular therapy : the journal of the American Society of Gene Therapy | 2014 | Yes |
| Oculopharyngeal muscular dystrophy: recent advances in the understanding of the molecular pathogenic mechanisms and treatment strategies. | Biochimica et biophysica acta | 2007 | Yes |
| Trehalose reduces aggregate formation and delays pathology in a transgenic mouse model of oculopharyngeal muscular dystrophy. | Human molecular genetics | 2006 | Yes |
Showing 5 of 10 publications.
Prevalence (12)
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 |
|---|---|---|---|---|---|
| Oculopharyngeal muscular dystrophy | Point prevalence | 1-9 / 100 000 | France | Not stated | Unspecified |
| Oculopharyngeal muscular dystrophy | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Unspecified |
| Oculopharyngeal muscular dystrophy | Point prevalence | >1 / 1000 | Specific population | Not stated | Unspecified |
| Oculopharyngeal muscular dystrophy | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Exact |
| Oculopharyngeal muscular dystrophy | Point prevalence | >1 / 1000 | Specific population | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Oculopharyngeal muscular dystrophy | 270 | Exact | Orphadata Science knowledge files, Product 1 |
| Oculopharyngeal muscular dystrophy | 270 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.