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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT05730842
What this trial is testing

Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers

Who this might be right for
Healthy VolunteerMuscular DystrophiesMuscular Disorders, Atrophic+5 more
Edgewise Therapeutics, Inc. 15
Early research (Phase 1)Study completedNCT04085666
What this trial is testing

Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria
Société des Produits Nestlé (SPN) 18
Not applicableLooking for participantsNCT06325488
What this trial is testing

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease

Who this might be right for
Fabry DiseaseChronic Kidney Diseases
Caroline Michaela Kistorp 60
Not applicableStudy completedNCT00004753
What this trial is testing

Long-Term Study of Cerebral Glucose Metabolism in Huntington's Disease

Who this might be right for
Huntington's Disease
National Center for Research Resources (NCRR) 90
Not applicableEnded earlyNCT01330277
What this trial is testing

Biomarkers for Hunter Syndrome

Who this might be right for
Hunter SyndromeMucopolysaccharidosis IIHunter's Syndrome, Mild Form+1 more
CENTOGENE GmbH Rostock 11
Early research (Phase 1)Study completedNCT04095273
What this trial is testing

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

Who this might be right for
Advanced Solid Tumors
Bayer 56
Large-scale testing (Phase 3)Study completedNCT03485677
What this trial is testing

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Who this might be right for
Gaucher's Disease Type IGaucher's Disease Type III
Sanofi 57
Not applicableNot Yet RecruitingNCT07208565
What this trial is testing

Endocrine Dysfunction in Pediatric Wilson's Disease

Who this might be right for
Wilson's Disease
Assiut University 30
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableLooking for participantsNCT06967831
What this trial is testing

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Who this might be right for
Leigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR)
Charite University, Berlin, Germany 80
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572
What this trial is testing

Gene Therapy for Children With CLN3 Batten Disease

Who this might be right for
CLN3Batten Disease
Neela Therapeutics 7
Not applicableActive Not RecruitingNCT07765472
What this trial is testing

Subclinical Myocardial Dysfunction in Children With Wilson's Disease

Who this might be right for
Wilson's DiseaseMyocardial DysfunctionChildren+3 more
Hebatullah Fawzy 72
Not applicableStudy completedNCT04910581
What this trial is testing

RTMS in Wilson Disease Dysarthria

Who this might be right for
Wilson Disease
Assistance Publique - Hôpitaux de Paris 18
Not applicableLooking for participantsNCT04708431
What this trial is testing

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

Who this might be right for
Androgen Insensitivity SyndromeMetabolic Parameters in AIS, CAIS, PAIS and MAISTumor Formation in AIS, CAIS, PAIS and MAIS+1 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 650
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145
What this trial is testing

Gene Therapy Study for Children With CLN5 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc. 6
Not applicableStudy completedNCT02740153
What this trial is testing

PCORI Urea Cycle Disorder Study

Who this might be right for
Urea Cycle Disorders
Children's National Research Institute 187
Large-scale testing (Phase 3)Study completedNCT00880100
What this trial is testing

Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)

Who this might be right for
Cystic FibrosisPancreatic Insufficiency
Forest Laboratories 49
Large-scale testing (Phase 3)Study completedNCT00268944
What this trial is testing

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 5
Not applicableStudy completedNCT00472732
What this trial is testing

Neurologic Injuries in Adults With Urea Cycle Disorders

Who this might be right for
Brain Diseases, Metabolic, InbornUrea Cycle DisorderOrnithine Transcarbamylase Deficiency
Andrea Gropman 46
Testing effectiveness (Phase 2)Study completedNCT03771898
What this trial is testing

Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 36
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