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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
Genzyme, a Sanofi CompanyNCT00567073
Observational
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adult…
Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more
University of Missouri-ColumbiaNCT07478172
Phase 1
A Single-Arm, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of AB-1009 in Adult Participants With Late Onset Pompe Disease (LOPD)
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult …
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio IncNCT07282847