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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableStudy completedNCT03620071
What this trial is testing

GoalKeeper: Intelligent Information Sharing for Children With Medical Complexity

Who this might be right for
Childhood CancerCerebral PalsyChronic Lung Disease+3 more
Stanford University 67
Large-scale testing (Phase 3)Active Not RecruitingNCT05208281
What this trial is testing

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 32
Not applicableUnknownNCT02859363
What this trial is testing

Fabry Disease in Cerebrovascular Disease

Who this might be right for
Fabry Disease
Chang Gung Memorial Hospital 700
Not applicableStudy completedNCT05330039
What this trial is testing

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Who this might be right for
Inborn Errors of MetabolismPropionic AciduriaMethylmalonic Aciduria+1 more
Emma Marie Caroline Slack 22
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40
Testing effectiveness (Phase 2)Study completedNCT03519711
What this trial is testing

PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

Who this might be right for
BH4 DeficiencyHyperphenylalaninemia
PTC Therapeutics 8
Large-scale testing (Phase 3)Study completedNCT01842841
What this trial is testing

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 5
Not applicableUnknownNCT06065605
What this trial is testing

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Who this might be right for
Fabry Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 40
Testing effectiveness (Phase 2)Ended earlyNCT04145037
What this trial is testing

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
AVROBIO 8
Not applicableStudy completedNCT02460445
What this trial is testing

Phlebotomy and Polycystic Ovary Syndrome

Who this might be right for
HyperandrogenismMetabolic Cardiovascular Syndrome
Manuel Luque Ramírez 37
Testing effectiveness (Phase 2)UnknownNCT04940572
What this trial is testing

Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome

Who this might be right for
Wolfram Syndrome
Centre d'Etude des Cellules Souches 23
Not applicableStudy completedNCT04580979
What this trial is testing

Natural History Study of FDXR Mutation-related Mitochondriopathy

Who this might be right for
Neurodegenerative Disease, HereditaryMitochondrial DiseasesOptic Atrophy
State University of New York at Buffalo 33
Early research (Phase 1)Study completedNCT00645879
What this trial is testing

Anaplerotic Therapy in Propionic Acidemia

Who this might be right for
Propionic Acidemia
Nicola Longo 3
Testing effectiveness (Phase 2)Study completedNCT00467935
What this trial is testing

Scotoma Reduction in AMD Patients Treated With Ranibizumab

Who this might be right for
Macular Degeneration
Retina Research Foundation 20
Not applicableUnknownNCT03590548
What this trial is testing

Clinical Audit of Managment of Gausher Disease in Children

Who this might be right for
Gaucher Disease
Yasmeen Abd Elbaset Hamzaa 1
Large-scale testing (Phase 3)Study completedNCT05218655
What this trial is testing

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Who this might be right for
Inherited Mitochondrial Disease
PTC Therapeutics 101
Not applicableStudy completedNCT05545774
What this trial is testing

Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients

Who this might be right for
Cystinosis
Assistance Publique - Hôpitaux de Paris 20
Not applicableEnded earlyNCT06308718
What this trial is testing

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Who this might be right for
Krabbe Disease
Forge Biologics, Inc 2
Not applicableStudy completedNCT02952482
What this trial is testing

Newborn Screening for Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
National Taiwan University Hospital 45,796
Not applicableUnknownNCT05301218
What this trial is testing

CAlcified Lesion Intervention Planning Steered by OCT.

Who this might be right for
Coronary Artery CalcificationAngioplasty
Institut Mutualiste Montsouris 140
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