Indication
Mucopolysaccharidosis type I
3 clinical trials
3 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
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersStatus: Terminated, Estimated PCD: 2016-04-01