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Results for “Lysosomal Disease”

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Showing 20 of 0 results

Not applicableStudy completedNCT00414583
What this trial is testing

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients

Who this might be right for
Cerebrovascular Accident
CENTOGENE GmbH Rostock 5,000
Testing effectiveness (Phase 2)Study completedNCT01304277
What this trial is testing

This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.

Who this might be right for
Fabry Disease
Shire 17
Not applicableStudy completedNCT01439607
What this trial is testing

Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease

Who this might be right for
Gaucher Disease
Assistance Publique - Hôpitaux de Paris 30
Not applicableEnded earlyNCT00787995
What this trial is testing

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Who this might be right for
MPS IV AMucopolysaccharidosis IVAMorquio A Syndrome
BioMarin Pharmaceutical 353
Not applicableUnknownNCT00871611
What this trial is testing

Viennese Prevalence Study of Anderson-Fabry Disease

Who this might be right for
Fabry DiseaseLeft Ventricular Hypertrophy
Medical University of Vienna 4,000
Not applicableEnded earlyNCT02232477
What this trial is testing

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Who this might be right for
Mucopolysaccharidosis ICognitive Decline
agnes chen 6
Testing effectiveness (Phase 2)Study completedNCT01301898
What this trial is testing

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

Who this might be right for
Mucopolysaccharidosis II
Green Cross Corporation 31
Not applicableLooking for participantsNCT07382128
What this trial is testing

Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes

Who this might be right for
HCM - Hypertrophic CardiomyopathyAnderson Fabry DiseaseCardiac Magnetic Resonance Imaging+1 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 250
Large-scale testing (Phase 3)Ended earlyNCT04840667
What this trial is testing

Replagal in Treatment-naïve Adults With Fabry Disease

Who this might be right for
Fabry Disease
Shire 17
Not applicableStudy completedNCT02719249
What this trial is testing

Fabry Disease Screening in ESRD Patients in West of France

Who this might be right for
ESRD
Rennes University Hospital 500
Early research (Phase 1)Study completedNCT01427517
What this trial is testing

Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

Who this might be right for
Parkinson's DiseaseGaucher's Disease
University of Minnesota 9
Not applicableEnded earlyNCT07494058
What this trial is testing

Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico

Who this might be right for
Lysosomal Disease
Takeda 232
Not applicableWithdrawnNCT01457443
What this trial is testing

Biomarker for Pompe Disease (BioPompe)

Who this might be right for
Cardiac DiseasesMuscular WeaknessHepato-splenomegaly+4 more
CENTOGENE GmbH Rostock
Large-scale testing (Phase 3)WithdrawnNCT02921620
What this trial is testing

Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease

Who this might be right for
Fabry Disease
Protalix
Testing effectiveness (Phase 2)Study completedNCT00051935
What this trial is testing

The Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 2
Large-scale testing (Phase 3)Study completedNCT01371825
What this trial is testing

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 9
Post-approval studies (Phase 4)Study completedNCT00486889
What this trial is testing

Growth and Development Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease
Genzyme, a Sanofi Company 12
Not applicableStudy completedNCT06007768
What this trial is testing

Autoimmune and Inflammatory Response Biomarkers in Fabry Disease

Who this might be right for
Fabry Disease
Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal 32
Not applicableStudy completedNCT01509768
What this trial is testing

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Who this might be right for
Sanfilippo Syndrome Type B
Shire 19
Testing effectiveness (Phase 2)Looking for participantsNCT07169279
What this trial is testing

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 77
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