Gaucher Disease, Type II (DBCOND0117120)

Identifiers

Synonyms

Type II Gaucher Disease / Gaucher's disease type II / Gaucher Disease, Type 2 / Type 2 Gaucher Disease / Acute neuronopathic Gaucher's disease (disorder)

Associated Data

Indicated Drugs and Targets

Not Available

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT06272149	An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease	No drug interventions	treatment	0	recruiting
NCT04411654	Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)	Methylprednisolone Prednisone Sirolimus	treatment	1 / 2	active_not_recruiting
NCT04532047	In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases	Alglucosidase alfa Elosulfase alfa Galsulfase Idursulfase Laronidase Sebelipase alfa Vestronidase alfa	treatment	1	recruiting
NCT04470713	Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2	No drug interventions	Not Available	Not Available	completed
NCT00244582	Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease	No drug interventions	Not Available	Not Available	completed