Buscar resultados de ensayos clínicos
Lysosomal Storage Diseases - 50 Studies Found
Estado | Estudiar |
Terminated |
Nombre del estudio: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases Condición:
Fecha: 2008-04-03 Intervenciones: Biological: ALD-101 A subpopulation of cord blood cells composed of cells that express a high level of t |
Recruiting |
Nombre del estudio: Biomarker for Patient With Cystinosis Disease or Highly Suspected for Cystinosis Disease Condición:
|
Terminated |
Nombre del estudio: Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I Condición:
|
Completed |
Nombre del estudio: A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I Condición:
|
Recruiting |
Nombre del estudio: Biomarker for Metachromatic Leukodystrophy Disease Condición:
Fecha: 2012-02-21 |
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:
|