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GM1 gangliosidosis type 1

Infantile GM1 gangliosidosisGangliosidosis Generalized GM1 Type 1
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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-4 of 4 results.
Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis
Status: Not yet recruiting
Last Changed: Apr 30, 2020
First Received: Mar 25, 2020
Disease(s): Morquio B Disease, GM1 Gangliosidosis Type III
Locations: BC Children's Hospital, Vancouver, British Columbia, Canada
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type I and Type II GM1 Gangliosidosis
Status: Recruiting
Last Changed: Oct 01, 2020
First Received: May 16, 2019
Disease(s): Lysosomal Diseases, Gangliosidosis, GM1
Intervention(s): AAV9-GLB1, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessmentwith ABR, Bone density scan (DEXA, Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake andextended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland, United States
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Status: Recruiting
Last Changed: Sep 30, 2020
First Received: Aug 01, 2019
Disease(s): GM1 Gangliosidosis
Locations: UCSF Benioff Children's Hospital Oakland, Oakland, California, United States
University of Minnesota, Minneapolis, Minnesota, United States
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
Montreal Children's Hospital Research Institute - McGill University, Montreal, Quebec, Canada
... and 4 other locations.
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Status: Recruiting
Last Changed: Jul 27, 2020
First Received: Jul 14, 2020
Disease(s): GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Locations: Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Mayo Clinic - Rochester, Rochester, Minnesota, United States
Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, Virginia, United States
UCL Cliniques Universitaires Saint-Luc, Bruxelles, Belgium
Hospital de Clínicas de Porto Alegre - HCPA, Porto Alegre, Brazil
... and 13 other locations.