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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableStudy completedNCT04318509
What this trial is testing

Market Research - Acceptability Study for a New PKU Protein Substitute

Who this might be right for
PKUPhenylketonuriasInborn Errors of Metabolism+1 more
Dr. Schär AG / SPA 8
Not applicableStudy completedNCT02851563
What this trial is testing

A Natural History Study of Canavan Disease

Who this might be right for
Canavan Disease
NYU Langone Health 21
Testing effectiveness (Phase 2)Study completedNCT03933163
What this trial is testing

Micronised Resveratrol as a Treatment for Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
Murdoch Childrens Research Institute 25
Not applicableUnknownNCT06169358
What this trial is testing

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy

Who this might be right for
Hypertrophic CardiomyopathyLeft Ventricular HypertrophyFabry Disease
Qilu Hospital of Shandong University 627
Large-scale testing (Phase 3)Study completedNCT06431113
What this trial is testing

Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on Pattern-electroretinogram in Glaucoma

Who this might be right for
GlaucomaNeuroprotection
Fondazione IRCCS Policlinico San Matteo di Pavia 40
Not applicableStudy completedNCT01165697
What this trial is testing

Establishment of Biomarkers for Fabry Disease

Who this might be right for
Fabry Disease
Ohio State University 4
Not applicableStudy completedNCT02763215
What this trial is testing

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

Who this might be right for
Wilson Disease
Alexion Pharmaceuticals, Inc. 64
Testing effectiveness (Phase 2)Study completedNCT00391625
What this trial is testing

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Who this might be right for
Gaucher Disease
Shire 10
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

Who this might be right for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
Not applicableStudy completedNCT01358474
What this trial is testing

Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism

Who this might be right for
Parkinson DiseaseGaucher DiseaseIdiopathic Rapid Eye Movement Sleep Disorder
University of Minnesota 56
Testing effectiveness (Phase 2)Study completedNCT01380743
What this trial is testing

Drug-drug Interaction Study

Who this might be right for
Pompe Disease
Amicus Therapeutics 25
Large-scale testing (Phase 3)Study completedNCT02797080
What this trial is testing

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
Amgen 38
Post-approval studies (Phase 4)Study completedNCT01410890
What this trial is testing

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD II)
Genzyme, a Sanofi Company 21
Not applicableStudy completedNCT04120168
What this trial is testing

Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease

Who this might be right for
Duchenne Muscular DystrophyPompe Disease (Late-onset)
Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition 590
Not applicableUnknownNCT01442350
What this trial is testing

Myocardial Affectation in Patients With Fabry Disease Without Phenotypic Manifestation. Diagnostic Value of Biomarkers

Who this might be right for
Fabry Disease, Cardiac VariantRight Ventricular Hypertrophy
Francisco Marín Ortuño 120
Early research (Phase 1)WithdrawnNCT04256655
What this trial is testing

Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients

Who this might be right for
Phenylketonurias
Société des Produits Nestlé (SPN)
Testing effectiveness (Phase 2)Study completedNCT00214500
What this trial is testing

AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 9
Testing effectiveness (Phase 2)Study completedNCT01801709
What this trial is testing

Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
Institut National de la Santé Et de la Recherche Médicale, France 5
Not applicableLooking for participantsNCT05239858
What this trial is testing

International Wilson's Disease Patient Registry (iWilson Registry)

Who this might be right for
Wilson's Disease
Orphalan 500
Not applicableEnrolling By InvitationNCT01803906
What this trial is testing

Tissue Sample Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DisordersMitochondrial DiseaseMelas+6 more
Columbia University 6,900
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