Gangliosidoses, GM2 clinical trials at UCSF
1 research study open to eligible people
Gangliosidoses, like GM2, are inherited conditions damaging brain tissues. UCSF tests an oral medication’s impact on GM2 gangliosidosis in children and teens. This trial follows a strict procedure with placebos to ensure precise comparison over 18 months.
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Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
open to eligible people ages 4 years and up
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-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis
Oakland, California and other locations
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