Gangliosidosis Overview
Learn About Gangliosidosis
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However, there may be experts who have treated this or similar conditions in our Find a Doctor section and research may be available in our Latest Advances section.
Cynthia Tifft is a Medical Genetics provider in Washington, Washington, D.c.. Dr. Tifft is rated as an Elite provider by MediFind in the treatment of Gangliosidosis. Her top areas of expertise are Sandhoff Disease, Gangliosidosis, GM1 Gangliosidosis, and Tay-Sachs Disease.
Massachusetts General Physicians Organization Inc
Florian Eichler is a Neurologist in Boston, Massachusetts. Dr. Eichler is rated as an Elite provider by MediFind in the treatment of Gangliosidosis. His top areas of expertise are Adrenoleukodystrophy (ALD), CACH Syndrome, Tay-Sachs Disease, Gangliosidosis, and Gastrostomy. Dr. Eichler is currently accepting new patients.
Jagdeep Walia practices in Kingston, Canada. Mr. Walia is rated as an Elite expert by MediFind in the treatment of Gangliosidosis. His top areas of expertise are Sandhoff Disease, Tay-Sachs Disease, Gangliosidosis, and Medium-Chain Acyl-CoA Dehydrogenase Deficiency.
Summary: 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 Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme. This could improve GM1 symptoms.