Clinical Research Directory
Browse clinical research sites, groups, and studies.
3 clinical studies listed.
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Tundra lists 3 Sly Syndrome clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT02171104
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Gender: All
Ages: Any - 55 Years
Updated: 2026-11-03
1 state
NCT03604835
Mucopolysaccharidosis VII Disease Monitoring Program
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
Gender: All
Updated: 2026-08-27
12 states
NCT03775174
Expanded Access to Mepsevii
Individual patient expanded access requests may be considered for patients who have no other treatment options
Gender: All
Updated: 2026-07-30