Clinical trials
Terminated Observational Lysosomal Storage Diseases 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 |
|---|---|
| 2002 | 1 |
| 2008 | 1 |
| 2009 | 1 |
| 2015 | 1 |
| 2019 | 2 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Terminated | 7 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| AVROBIO | 1 |
| Baylor Research Institute | 1 |
| BioMarin Pharmaceutical | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Forge Biologics, Inc | 1 |
| Neurogene Inc. | 1 |
| Shire | 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 |
|---|---|
| Lysosomal Storage Diseases | 7 |
| Demyelinating Diseases | 2 |
| Agenesis of Corpus Callosum | 1 |
| Aspartylglucosaminuria | 1 |
| Brain Diseases | 1 |
| Brain Diseases, Metabolic, Inborn | 1 |
| Central Nervous System Diseases | 1 |
| Corneal Opacity | 1 |
| Fabry Disease | 1 |
| Genetic Diseases, Inborn | 1 |
Trials
Showing 1 to 7 of 7. Each trial links to its page on Pharma Observatory.
Recently changed trials
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