Clinical trials
Terminated Lysosomal Storage Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
17 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2002 | 1 |
| 2005 | 1 |
| 2006 | 2 |
| 2008 | 3 |
| 2009 | 1 |
| 2012 | 2 |
| 2015 | 1 |
| 2018 | 1 |
| 2019 | 2 |
| 2021 | 2 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Terminated | 17 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 3 |
| Genzyme, a Sanofi Company | 2 |
| Patricia I. Dickson, M.D. | 2 |
| AVROBIO | 1 |
| Aldagen | 1 |
| Baylor Research Institute | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Forge Biologics, Inc | 1 |
| LYSOGENE | 1 |
| Neurogene 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 |
|---|---|
| Lysosomal Storage Diseases | 17 |
| Fabry Disease | 3 |
| Mucopolysaccharidoses | 3 |
| Mucopolysaccharidosis I | 3 |
| Mucopolysaccharidosis IV | 3 |
| Demyelinating Diseases | 2 |
| Gangliosidosis, GM1 | 2 |
| Leukodystrophy, Globoid Cell | 2 |
| Leukodystrophy, Metachromatic | 2 |
| Metabolism, Inborn Errors | 2 |
Trials
Showing 1 to 17 of 17. 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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