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Lysosomal Storage Diseases, Nervous System - 25 Studies Found
Estado | Estudiar |
Recruiting |
Nombre del estudio: Biomarker for Metachromatic Leukodystrophy Disease Condición:
Fecha: 2012-02-21 |
Recruiting |
Nombre del estudio: Biomarker for Pompe Disease Condición:
Fecha: 2011-10-21 |
Completed |
Nombre del estudio: Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease Condición: Enzyme Replacement Therapy in Pompe Disease Fecha: 2017-04-30 |
Not yet recruiting |
Nombre del estudio: Search for Serum/Plasma Biomarkers in Pompe's Disease Condición:
Fecha: 2017-01-27 Intervenciones: Drug: Enzyme Replacement Agent Patients will be treated following the decision of their doctors. The inv |
Not yet recruiting |
Nombre del estudio: 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 Condición: Glycogen Storage Disease Type II-Pompe's Disease Fecha: 2016-12-20 Intervenciones:
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Recruiting |
Nombre del estudio: Pompe Telemedicine Developmental Study Condición:
Fecha: 2016-06-22 |
Completed |
Nombre del estudio: Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth Condición: Pompe's Disease Fecha: 2016-09-06 Intervenciones: Other: observational (no intervention) |
Completed |
Nombre del estudio: Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana Condición: Pompe's Disease Fecha: 2016-09-06 Intervenciones: Other: observational |
Not yet recruiting |
Nombre del estudio: VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease Condición: Pompe Disease Fecha: 2016-09-08 Intervenciones:
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Recruiting |
Nombre del estudio: Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices Condición: Pompe Disease Fecha: 2016-05-12 |