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MPS VII
Clinical trial pipeline · Data from ClinicalTrials.gov
See which MPS VII trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which MPS VII trials you may qualify forThe objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersens…
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and…
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collect…
Individual patient expanded access requests may be considered for patients who have no other treatment options
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-rel…
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that prov…