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

~Estimated

~$10 – $8,750

AskBio IncNCT07282847

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

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

~Estimated

~$10 – $6,250

University of Missouri-ColumbiaNCT07478172