Lysosomal Storage Diseases, Nervous System - 15 Studies Found Completed : Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease : Enzyme Replacement Therapy in Pompe Disease : 2017-04-30 Not yet recruiting : Search for Serum/Plasma Biomarkers in Pompe's Disease : Late Onset Pompe Disease Pompe Disease : 2017-01-27 : Drug: Enzyme Replacement Agent Patients will be treated following the decision of their doctors. The inv Not yet recruiting : A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa : Glycogen Storage Disease Type II-Pompe's Disease : 2016-12-20 : Drug: GZ402666 Pharmaceutical Recruiting : Pompe Telemedicine Developmental Study : Pompe Disease Glycogen Storage Disease II : 2016-06-22 Completed : Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth : Pompe's Disease : 2016-09-06 : Other: observational (no intervention) Completed : Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana : Pompe's Disease : 2016-09-06 : Other: observational Not yet recruiting : VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease : Pompe Disease : 2016-09-08 : Drug: VAL-1221 1 mg/kg VAL-122 Completed : Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices : Pompe Disease : 2016-05-12 Completed : Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study) : Pompe Disease : 2016-06-20 : Drug: glucosidase alfa Long-term use in an observational study of licenced drug Completed : Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease : Glycogen Storage Disease Type II-Pompe's Disease : 2016-05-23 : Drug: GZ402666 Next >>>
