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