Clinical trials
Lysosomal Storage Diseases trials in Brazil
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
16 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2004 | 1 |
| 2007 | 1 |
| 2008 | 2 |
| 2011 | 2 |
| 2014 | 1 |
| 2015 | 2 |
| 2018 | 2 |
| 2019 | 1 |
| 2020 | 1 |
| 2021 | 1 |
| 2022 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Terminated | 4 |
| Active, not recruiting | 2 |
| Suspended | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Shire | 4 |
| BioMarin Pharmaceutical | 3 |
| AVROBIO | 2 |
| Gemma Biotherapeutics | 2 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Azafaros B.V. | 1 |
| REGENXBIO Inc. | 1 |
| Ultragenyx Pharmaceutical Inc | 1 |
| University of Pennsylvania | 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 | 16 |
| Mucopolysaccharidosis II | 4 |
| Sudden Infant Death | 4 |
| Gangliosidosis, GM1 | 3 |
| Genetic Diseases, Inborn | 3 |
| Metabolic Diseases | 3 |
| Mucopolysaccharidosis IV | 3 |
| Rare Diseases | 3 |
| Fabry Disease | 2 |
| Lipid Metabolism Disorders | 2 |
Trials
Showing 51 to 16 of 16. Each trial links to its page on Pharma Observatory.
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