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3 recruiting studies matching your filters
Phase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety an…
MPS IMPS IIMPS IVA+6 more
University of California, San FranciscoNCT04532047
Phase 2
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical IncNCT02716246
Longitudinal Study of Neurodegenerative Disorders
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 understandi…
MLDKrabbe DiseaseALD+24 more
University of PittsburghNCT03333200