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Lysosomal Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Lysosomal Diseases trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme r…
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other…
The purpose of this study is to better understand the natural history, clinical outcomes, and biological features of Gaucher disease in patients receiving stand…
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late…
The recovery of transit after surgery is an important parameter in postoperative evaluation. It generally reflects simple postoperative outcomes and allows the…
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
This is a Post Trial Access program for patients with Fabry Disease who previously participated in the ID-069A302 (MODIFY OLE) trial and who were still enrolled…
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their prim…