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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT04776824
What this trial is testing

Swiss Cardiac Amyloidosis REgistry (Swiss-CARE)

Who this might be right for
Amyloid Cardiomyopathy
Insel Gruppe AG, University Hospital Bern 300
Testing effectiveness (Phase 2)UnknownNCT02663024
What this trial is testing

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
Green Cross Corporation 20
Not applicableLooking for participantsNCT07435168
What this trial is testing

CHEST-CA: Study of Chest Pain and Hidden Cardiac Amyloidosis

Who this might be right for
Cardiac AmyloidosisChest Pain
Rigshospitalet, Denmark 300
Large-scale testing (Phase 3)Ended earlyNCT00545597
What this trial is testing

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

Who this might be right for
AdrenomyeloneuropathyAdrenoleukodystrophy
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 240
Not applicableUnknownNCT06260982
What this trial is testing

Cognitive Disorders in Hereditary Spastic Paraplegia Type 4

Who this might be right for
Spastic Paraplegia
Central Hospital, Nancy, France 30
Early research (Phase 1)Looking for participantsNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Not applicableStudy completedNCT03911089
What this trial is testing

A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

Who this might be right for
Urea Cycle DisorderInborn Errors of Metabolism
Nutricia Research 3
Not applicableLooking for participantsNCT06573723
What this trial is testing

Institutional Registry of Rare Diseases

Who this might be right for
Rare DiseasesAmyloidosisSarcoidosis+23 more
Hospital Italiano de Buenos Aires 380
Not applicableLooking for participantsNCT07766135
What this trial is testing

Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis

Who this might be right for
Transthyretin (TTR) Amyloid CardiomyopathyCardiac AmyloidosisCardiomyopathies+2 more
University of Messina 70
Not applicableStudy completedNCT02354651
What this trial is testing

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 9
Not applicableNot Yet RecruitingNCT07315126
What this trial is testing

Reliability of Transcutaneous Bilirubin Measurement According to the Skin Colour of Newborns

Who this might be right for
Neonatal Jaundice
Assistance Publique - Hôpitaux de Paris 510
Not applicableStudy completedNCT04201418
What this trial is testing

Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Who this might be right for
Hereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathy
Alnylam Pharmaceuticals 67
Early research (Phase 1)Study completedNCT00692926
What this trial is testing

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

Who this might be right for
MDSAnemia, AplasticInborn Errors of Metabolism+2 more
Joanne Kurtzberg, MD 37
Not applicableLooking for participantsNCT06877715
What this trial is testing

Autistic Symptomatology and Sensory Profile in Children With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
University Hospital, Toulouse 75
Not applicableWithdrawnNCT02462213
What this trial is testing

Prospective Identification of Cardiac Amyloidosis by Cardiac Magnetic Resonance Imaging

Who this might be right for
AmyloidosisParaproteinemiasCardiomyopathies
University of Cincinnati
Not applicableActive Not RecruitingNCT03090789
What this trial is testing

FA Clinical Outcome Measures

Who this might be right for
Friedreich AtaxiaNeuro-Degenerative Disease
Friedreich's Ataxia Research Alliance 2,000
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Large-scale testing (Phase 3)Study completedNCT05099640
What this trial is testing

PTC923 in Participants With Phenylketonuria

Who this might be right for
Phenylketonuria
PTC Therapeutics 157
Not applicableStudy completedNCT06201832
What this trial is testing

Cardiac Amyloidosis in HFpEF Tunisian Patients

Who this might be right for
Cardiac AmyloidosisHeart Failure With Preserved Ejection FractionEchocardiography+4 more
University Tunis El Manar 87
Testing effectiveness (Phase 2)Looking for participantsNCT07371663
What this trial is testing

An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors

Who this might be right for
Solid CancersNSCLC (Advanced Non-small Cell Lung Cancer)Gastric (Stomach) Cancer+2 more
Beijing Tide Pharmaceutical Co., Ltd 266
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