Gangliosidosis
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Gangliosidosis Overview

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Learn About Gangliosidosis

Condition 101 content is not available at this time, but we are continually updating the site. Please check back.

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.

Who are the top Gangliosidosis Local Doctors?
Elite in Gangliosidosis
Dr. Cynthia J. Tifft
Medical Genetics
Elite in Gangliosidosis
Dr. Cynthia J. Tifft
Medical Genetics
111 Michigan Ave Nw, 
Washington, DC 
202-884-2187
Languages Spoken:
English

Cynthia Tifft is a Medical Genetics provider practicing medicine in Washington, Washington, D.c.. Dr. Tifft is rated as an Elite provider by MediFind in the treatment of Gangliosidosis. She is also highly rated in 11 other conditions, according to our data. Her clinical expertise encompasses Sandhoff Disease, Gangliosidosis, GM1 Gangliosidosis, and Tay-Sachs Disease.

Elite in Gangliosidosis
Jagdeep S. Walia
Elite in Gangliosidosis
Jagdeep S. Walia
Kingston, ON, CA 

Jagdeep Walia practices practicing medicine in Kingston, Canada. Mr. Walia is rated as an Elite expert by MediFind in the treatment of Gangliosidosis. He is also highly rated in 6 other conditions, according to our data. His clinical expertise encompasses Sandhoff Disease, Tay-Sachs Disease, Gangliosidosis, and Medium-Chain Acyl-CoA Dehydrogenase Deficiency.

 
 
 
 
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Elite in Gangliosidosis
Dr. Florian Eichler
Pediatric Neurology
Elite in Gangliosidosis
Dr. Florian Eichler
Pediatric Neurology

Massachusetts General Hospital

55 Fruit St, 
Boston, MA 
617-726-6093
Languages Spoken:
English
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Florian Eichler is a Pediatric Neurologist practicing medicine in Boston, Massachusetts. Dr. Eichler is rated as an Elite provider by MediFind in the treatment of Gangliosidosis. He is also highly rated in 25 other conditions, according to our data. His clinical expertise encompasses Adrenoleukodystrophy (ALD), CACH Syndrome, Tay-Sachs Disease, Gangliosidosis, and Gastrostomy.

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What are the latest Gangliosidosis Clinical Trials?
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
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
Enrollment Status: Recruiting
Publish Date: April 15, 2026
Intervention Type: Drug
Study Phase: Phase 3

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

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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
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
Enrollment Status: Recruiting
Publish Date: April 15, 2026
Intervention Type: Drug
Study Phase: Phase 3

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 GM1 gangliosidosis or GM2 gangliosidosis

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