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Gaucher disease type 2

Acute neuronopathic Gaucher's disease
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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-3 of 3 results.
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 14, 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.
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Status: Not yet recruiting
Last Changed: Jun 04, 2020
First Received: Jun 02, 2020
Disease(s): Gaucher Disease, Type 2
Intervention(s): PR001
Locations: UCSF Benioff Children's Hospital, 5700 Martin Luther King Jr Way, Oakland, California, United States
University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue, Minneapolis, Minnesota, United States
NYU Medical Center, 550 1st Avenue, New York, New York, United States
Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh, Pennsylvania, United States
Phase 1/2 Lentiviral Vector Gene Therapy - The GuardOne Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
Status: Recruiting
Last Changed: Feb 17, 2020
First Received: Oct 30, 2019
Disease(s): Gaucher Disease
Intervention(s): AVR-RD-02
Locations: Royal Melbourne Hospital, Melbourne, Parkville, VIC, Australia
University of Calgary, Calgary, Calgary Alberta, Canada