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GM1 Gangliosidosis
Clinical trial pipeline · Data from ClinicalTrials.gov
See which GM1 Gangliosidosis trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which GM1 Gangliosidosis trials may be worth asking aboutNorth America
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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…
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…
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galacto…
Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantitat…
This is a prospective observational multi-country, multi-center study of a large real-world cohort of first line (1L) epithelial ovarian cancer patients, expose…
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 understandin…
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that prov…
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…