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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableUnknownNCT04302194
What this trial is testing

Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.

Who this might be right for
Phenylketonurias
Assiut University 50
Not applicableStudy completedNCT05046379
What this trial is testing

Studying Lipids as Potential Biomarkers in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Vastra Gotaland Region 108
Not applicableEnded earlyNCT05917327
What this trial is testing

Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease

Who this might be right for
Hepatolenticular Degeneration; Wilson
Mexbrain 1
Not applicableUnknownNCT01898325
What this trial is testing

Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan

Who this might be right for
Fibrosis
Rambam Health Care Campus 40
Not applicableStudy completedNCT04948138
What this trial is testing

Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) Syndrome

Who this might be right for
MELAS Syndrome
Jesús González de la Aleja Tejera 9
Large-scale testing (Phase 3)Ended earlyNCT03879655
What this trial is testing

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 2
Not applicableStudy completedNCT02051049
What this trial is testing

Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)

Who this might be right for
Urea Cycle DisordersCrigler Najjar Syndrome
Cellaion SA 9
Large-scale testing (Phase 3)Study completedNCT02795676
What this trial is testing

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

Who this might be right for
Fabry Disease
Protalix 78
Not applicableEnded earlyNCT02246491
What this trial is testing

Cell-Based Approaches For Modeling and Treating Ataxia-Telangiectasia

Who this might be right for
Ataxia-Telangiectasia (A-T)
Johns Hopkins University 6
Large-scale testing (Phase 3)Study completedNCT00478647
What this trial is testing

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Who this might be right for
Gaucher Disease
Shire 40
Not applicableLooking for participantsNCT05231876
What this trial is testing

French Wilson Disease Registry

Who this might be right for
Wilson Disease
Fondation Ophtalmologique Adolphe de Rothschild 1,000
Not applicableStudy completedNCT05028764
What this trial is testing

Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

Who this might be right for
Friedreich AtaxiaHealthy Volunteers
Larimar Therapeutics, Inc. 60
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 applicableLooking for participantsNCT06941532
What this trial is testing

GMP Powdered Substitutes in PKU and TYR

Who this might be right for
PhenylketonuriaTyrosinemia
Nutricia UK Ltd 40
Not applicableUnknownNCT05696912
What this trial is testing

Functional Tests to Resolve Unsolved Rare Diseases. Rares.

Who this might be right for
Intellectual DisabilityRubinstein-Taybi SyndromeCystic Fibrosis+4 more
University Hospital, Bordeaux 50
Not applicableApproved For MarketingNCT00074919
What this trial is testing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company
Testing effectiveness (Phase 2)Study completedNCT00075244
What this trial is testing

Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableActive Not RecruitingNCT06495567
What this trial is testing

Proof of Concept Creatine Supplementation for Homocystinuria Study

Who this might be right for
Homocystinuria
University of British Columbia 9
Not applicableStudy completedNCT07046507
What this trial is testing

The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease

Who this might be right for
Wilson Disease
Royal Surrey County Hospital NHS Foundation Trust 10
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
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