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Mucopolysaccharidosis I (MPS I)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis I (MPS I) trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis I (MPS I) trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
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…
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandin…
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their prim…
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector…