Clinical trials
Recruiting Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
6 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2010 | 1 |
| 2022 | 1 |
| 2025 | 1 |
| 2026 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 6 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Arrowhead Pharmaceuticals | 1 |
| Children's Hospital of Philadelphia | 1 |
| Dyne Therapeutics | 1 |
| JCR Pharmaceuticals Co., Ltd. | 1 |
| Johns Hopkins University | 1 |
| National Human Genome Research Institute (NHGRI) | 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 |
|---|---|
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 6 |
| Genetic Diseases, Inborn | 4 |
| Congenital Abnormalities | 2 |
| Metabolism, Inborn Errors | 2 |
| Brain Diseases, Metabolic, Inborn | 1 |
| Carbohydrate Metabolism, Inborn Errors | 1 |
| Dyslipidemias | 1 |
| Fucosidosis | 1 |
| Genetic Diseases, X-Linked | 1 |
| Hernia | 1 |
Trials
Showing 1 to 6 of 6. 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
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