Clinical trials
Recruiting Phase 3 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
7 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2022 | 1 |
| 2024 | 1 |
| 2025 | 1 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 7 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Arrowhead Pharmaceuticals | 1 |
| Avidity Biosciences, Inc. | 1 |
| Chiesi Farmaceutici S.p.A. | 1 |
| Dyne Therapeutics | 1 |
| Grifols Therapeutics LLC | 1 |
| Intellia Therapeutics | 1 |
| Vertex Pharmaceuticals Incorporated | 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 | 7 |
| Neuromuscular Diseases | 3 |
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 2 |
| Metabolic Diseases | 2 |
| Metabolism, Inborn Errors | 2 |
| Muscular Dystrophies | 2 |
| Muscular Dystrophy, Duchenne | 2 |
| Myotonic Dystrophy | 2 |
| Nervous System Diseases | 2 |
| Amyloid Neuropathies | 1 |
Trials
Showing 1 to 7 of 7. 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.
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