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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Looking for participantsNCT06904261
What this trial is testing

Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Who this might be right for
Fabry Disease
Amicus Therapeutics 8
Testing effectiveness (Phase 2)Study completedNCT02107846
What this trial is testing

An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

Who this might be right for
Gaucher Disease
Protalix 10
Testing effectiveness (Phase 2)Study completedNCT01016366
What this trial is testing

Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
H. Lundbeck A/S 36
Not applicableStudy completedNCT04845958
What this trial is testing

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

Who this might be right for
Gaucher Disease, SplenomegalyAcid SphingoMyelinase Deficiency
Sanofi 60
Not applicableActive Not RecruitingNCT04798378
What this trial is testing

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

Who this might be right for
Neurologic DiseasesHemiparesisQuadriplegia+11 more
Thomas Jefferson University 20
Not applicableStudy completedNCT02594917
What this trial is testing

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

Who this might be right for
Iron-sulfur Cluster DeficiencyPulmonary HypertensionFriedreich Ataxia
University of Pittsburgh 13
Testing effectiveness (Phase 2)Study completedNCT01560182
What this trial is testing

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseaseMetachromatic Leukodystrophy
Orchard Therapeutics 20
Not applicableNot Yet RecruitingNCT07529860
What this trial is testing

AI-based Echocardiography for Detection of Cardiac Amyloidosis

Who this might be right for
Cardiac AmyloidosisHeart Failure With Preserved Ejection Fraction (HFPEF)Left Ventricular Hypertrophy
Germans Trias i Pujol Hospital 200
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
Not applicableNot Yet RecruitingNCT03725670
What this trial is testing

Direct Lentiviral Injection Gene Therapy for MLD

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shenzhen Geno-Immune Medical Institute 10
Not applicableStudy completedNCT02468570
What this trial is testing

Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 9
Not applicableStudy completedNCT01002469
What this trial is testing

Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity Assessment

Who this might be right for
Urea Cycle Disorders
Cytonet GmbH & Co. KG 10
Not applicableStudy completedNCT03299829
What this trial is testing

A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease Patients

Who this might be right for
Trientine Treatment for Wilson's Disease
Excelsior 48
Not applicableStudy completedNCT04050137
What this trial is testing

Therapeutic Exercise to Treat Neuropathic Pain

Who this might be right for
Gaucher DiseaseFabry Disease
Universidad de Zaragoza 5
Not applicableStudy completedNCT01743196
What this trial is testing

Folate Metabolism in Normal Weight and Obese Women of Child-bearing Age

Who this might be right for
ObesityNeural Tube Defects
University of Georgia 32
Not applicableLooking for participantsNCT07701694
What this trial is testing

Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task

Who this might be right for
Urea Cycle Disorder
St. Jude Children's Research Hospital 60
Large-scale testing (Phase 3)Study completedNCT01896102
What this trial is testing

The Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 32
Large-scale testing (Phase 3)Active Not RecruitingNCT06673056
What this trial is testing

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

Who this might be right for
Ataxia-TelangiectasiaAtaxia-Telangiectasia (A-T)
IntraBio Inc 60
Not applicableLooking for participantsNCT07484945
What this trial is testing

Multiomics Approach in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
University Hospital, Tours 149
Not applicableStudy completedNCT03548480
What this trial is testing

Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

Who this might be right for
Prader-Willi Syndrome
Fundació Sant Joan de Déu 39
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