Review clinical trials related to MPS VII. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.
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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.
Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic o... [+2]
Concurrent participation in other pharmaceutical company-sponsored interventiona...
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.
Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation [+4]
Fetuses with a concurrent severe structural anomaly [+10]