Clinical trials
Expanded access Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
8 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
No trials to chart.
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Approved for marketing | 3 |
| No longer available | 3 |
| Available | 2 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Akcea Therapeutics | 1 |
| Alnylam Pharmaceuticals | 1 |
| Avidity Biosciences, Inc. | 1 |
| Christopher Dvorak | 1 |
| GenSight Biologics | 1 |
| Ionis Pharmaceuticals, Inc. | 1 |
| NS Pharma, Inc. | 1 |
| RiboNova Inc. | 1 |
Top conditions
MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Genetic Diseases, Inborn | 8 |
| Metabolic Diseases | 3 |
| Metabolism, Inborn Errors | 3 |
| Muscular Diseases | 3 |
| Musculoskeletal Diseases | 3 |
| Dyslipidemias | 2 |
| Genetic Diseases, X-Linked | 2 |
| Hyperlipidemias | 2 |
| Hyperlipoproteinemia Type I | 2 |
| Hyperlipoproteinemias | 2 |
Trials
Showing 1 to 8 of 8. Each trial links to its page on Pharma Observatory.
Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
No changes to these trials in the last 7 days.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.