Clinical Trial Update: A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 [Phase 3]

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Summary

Phase 3 trial to evaluate the safety, efficacy, pharmacokinetics, pharmacodynamics and immunogenicity of cipaglucosidase alfa with miglustat in infants and youn…

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Diseases / indications

• Glycogen Storage Disease Type Ii

Drugs mentioned

• Miglustat (miglustat)
• cipaglucosidase alfa (cipaglucosidase alfa)

Snippet

Sponsor: Amicus Therapeutics | Conditions: Glycogen Storage Disease Type II Infantile Onset | Phase 3 | Status: RECRUITING