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Lysosomal Storage Diseases, Nervous System - 15 Studies Found

Status Study
Completed Study Name: Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease
Condition: Enzyme Replacement Therapy in Pompe Disease
Date: 2017-04-30
Not yet recruiting Study Name: Search for Serum/Plasma Biomarkers in Pompe's Disease
Condition:
  • Late Onset Pompe Disease
  • Pompe Disease

Date: 2017-01-27
Interventions: Drug: Enzyme Replacement Agent Patients will be treated following the decision of their doctors. The inv
Not yet recruiting Study Name: 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
Condition: Glycogen Storage Disease Type II-Pompe's Disease
Date: 2016-12-20
Interventions:
  • Drug: GZ402666 Pharmaceutical

Recruiting Study Name: Pompe Telemedicine Developmental Study
Condition:
  • Pompe Disease
  • Glycogen Storage Disease II

Date: 2016-06-22
Completed Study Name: Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth
Condition: Pompe's Disease
Date: 2016-09-06
Interventions: Other: observational (no intervention)
Completed Study Name: Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana
Condition: Pompe's Disease
Date: 2016-09-06
Interventions: Other: observational
Not yet recruiting Study Name: VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease
Condition: Pompe Disease
Date: 2016-09-08
Interventions:
  • Drug: VAL-1221 1 mg/kg VAL-122
Recruiting Study Name: Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices
Condition: Pompe Disease
Date: 2016-05-12
Recruiting Study Name: Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study)
Condition: Pompe Disease
Date: 2016-06-20
Interventions: Drug: glucosidase alfa Long-term use in an observational study of licenced drug
Recruiting Study Name: 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
Condition: Glycogen Storage Disease Type II-Pompe's Disease
Date: 2016-05-23
Interventions:
  • Drug: GZ402666

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