19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 899 Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

  
1 trial found
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agemin
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1NCT05324943
(ClinicalTrials.gov)
April 15, 202228/2/2022A Gene Therapy Study in Patients With Gaucher Disease Type 1A Phase 1/2, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)Gaucher Disease, Type 1Genetic: FLT201Freeline TherapeuticsNULLRecruiting18 YearsN/AAll18Phase 1/Phase 2Argentina;Brazil;Germany;Israel;Paraguay;Spain;United Kingdom;Italy;Tunisia