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Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Conditions:   GM1 Gangliosidosis;   GM2 Gangliosidosis;   Gaucher Disease, Type 2;   Tay-Sachs Disease;   AB Variant Gangliosidosis GM2;   Sandhoff Disease Intervention:   Sponsor:   Idorsia Pharmaceuticals Ltd. Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - July 14, 2020 Category: Research Source Type: clinical trials

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Condition:   Gaucher Disease, Type 2 Intervention:   Biological: PR001 Sponsor:   Prevail Therapeutics Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - June 2, 2020 Category: Research Source Type: clinical trials

Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Condition:   Gaucher Disease, Type 2 Intervention:   Biological: PR001 Sponsor:   Prevail Therapeutics Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - June 2, 2020 Category: Research Source Type: clinical trials