Gangliosidosis Recruiting Phase 1 / 2 Trials for Sirolimus (DB00877)

IndicationStatusPhase
DBCOND0071658 (Gangliosidosis)Recruiting1 / 2
clinicaltrials.gov IdentifierTitlePurposeDrugs
NCT03952637A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type I and Type II GM1 GangliosidosisTreatment