Clinical trials
Phase 2 Lysosomal Storage Diseases trials in the United Kingdom
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 |
|---|---|
| 2011 | 2 |
| 2012 | 2 |
| 2014 | 2 |
| 2023 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 4 |
| Terminated | 2 |
| Recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 3 |
| Alexion Pharmaceuticals, Inc. | 2 |
| Rocket Pharmaceuticals 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 |
| Mucopolysaccharidosis IV | 3 |
| Cholesterol Ester Storage Disease | 2 |
| Wolman Disease | 2 |
| Cardiomyopathies | 1 |
| Cardiomyopathy, Hypertrophic | 1 |
| Genetic Diseases, Inborn | 1 |
| Glycogen Storage Disease Type IIb | 1 |
| Hypertrophy, Left Ventricular | 1 |
| Infant, Newborn, Diseases | 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.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.