Recent studies have has shown that magnetic resonance spectroscopy (MRS) can provide validated neuronal markers in patients with Type 1 GD (GD1) who are on sta…

Gaucher Disease, Type 3

~Estimated

~$10 – $750

University of MinnesotaNCT05586243

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

~Estimated

~$10 – $5,625

University of California, San FranciscoNCT04532047