miglustat 2

Trials studying miglustat 2

• —A Global Prospective Observational Registry of Patients With Pompe Diseaserecruitingn=500
• phase3A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18recruitingn=36
• phase3Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutationcompletedn=16
• phase3ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDactive not recruitingn=21
• phase3A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Diseasecompletedn=125
• phase2An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Diseasecompletedn=6
• phase4Safety and Efficacy of Miglustat in Chinese NPC Patientscompletedn=17
• phase2Miglustat in Niemann-Pick Type C Diseasecompletedn=29