Lysosomal Storage Diseases, Nervous System - 25 Studies Found
Recruiting |
: Biomarker for Metachromatic Leukodystrophy Disease :
: 2012-02-21 |
Recruiting |
: Biomarker for Pompe Disease :
: 2011-10-21 |
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 :
: 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 :
|
Recruiting |
: Pompe Telemedicine Developmental Study :
: 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 :
|
Recruiting |
: Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices : Pompe Disease : 2016-05-12 |