Lysosomal Storage Diseases - 27 Studies Found

Terminated : Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
:
  • Mucopolysaccharidosis I
  • Lysosomal Storage Diseases
  • : 2005-09-19
    : Drug: laronidase 0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per mont
Completed : Magnetic Stimulation of the Human Nervous System
:
  • Demyelinating Disease
  • Healthy
  • Lysoso
    : 1999-11-03
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
:
  • Late Onset Pompe Disease
  • Pompe 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
:
  • Drug: GZ402666 Pharmaceutical

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

: 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
:
  • Drug: VAL-1221 1 mg/kg VAL-122
Recruiting : Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices
: Pompe Disease
: 2016-05-12
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