Indication
Hurler syndrome
4 clinical trials
10 products
Clinical trial
A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme® (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed InfantsStatus: Terminated, Estimated PCD: 2022-12-21
Product
LaronidaseProduct
RGX-111Clinical trial
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type IStatus: Active (not recruiting), Estimated PCD: 2023-03-01
Clinical trial
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGStatus: Recruiting, Estimated PCD: 2025-07-14
Product
Stem Cell TransplantationProduct
IMD Preparative RegimenProduct
OsteopetrosisProduct
CALD SR-AProduct
cALD SR-BProduct
cALD HR-DClinical trial
PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersStatus: Completed, Estimated PCD: 2022-02-17
Product
Thiotepa