Browse Studies — Arctuva

Pompe Disease Registry

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to tra…

Glycogen Storage Disease Type IIPompe Disease

Estimated

~$10 – $1,125

Genzyme, a Sanofi CompanyNCT00231400

A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease

This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant wom…

Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency

Estimated

~$10 – $1,125

Genzyme, a Sanofi CompanyNCT00567073

Phase 3

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ER…

Glycogen Storage Disease Type II Infantile Onset

Estimated

~$10 – $8,750

Amicus TherapeuticsNCT04808505