Clinical trials
Recruiting Genetic Diseases, Inborn trials in Australia
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
9 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2022 | 1 |
| 2023 | 1 |
| 2024 | 2 |
| 2025 | 1 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 9 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Murdoch Childrens Research Institute | 2 |
| 4D Molecular Therapeutics | 1 |
| AIRNA Corporation | 1 |
| Avidity Biosciences, Inc. | 1 |
| BioMarin Pharmaceutical | 1 |
| Friedreich's Ataxia Research Alliance | 1 |
| Neuroscience Research Australia | 1 |
| QED Therapeutics, a BridgeBio company | 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 | 9 |
| Musculoskeletal Diseases | 3 |
| Bone Diseases | 2 |
| Infant, Newborn, Diseases | 2 |
| Nervous System Diseases | 2 |
| Osteochondrodysplasias | 2 |
| Achondroplasia | 1 |
| Apraxias | 1 |
| Ataxia | 1 |
| Cardiovascular Diseases | 1 |
Trials
Showing 1 to 9 of 9. 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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