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Gangliosidosis GM1 Type 3

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Clinical Trials

This information is provided by ClinicalTrials.gov

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Longitudinal Study of Neurodegenerative Disorders
Status: Recruiting
Last Changed: Dec 05, 2018
First Received: Nov 06, 2017
Disease(s): MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Intervention(s): Palliative Care, Hematopoetic Stem Cell Transplantation
Locations: The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States