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Results for “Adult Refsum Disease”

10 trials

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Showing 10 of 10 results

Not applicableLooking for participantsNCT02435940
What this trial is testing

Inherited Retinal Degenerative Disease Registry

Who this might be right for
Eye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more
Foundation Fighting Blindness 20,000
Large-scale testing (Phase 3)Study completedNCT01838941
What this trial is testing

Betaine and Peroxisome Biogenesis Disorders

Who this might be right for
Peroxisome Biogenesis Disorders
McGill University Health Centre/Research Institute of the McGill University Health Centre 12
Not applicableEnded earlyNCT00004442
What this trial is testing

Study of Bile Acids in Patients With Peroxisomal Disorders

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more
University of Cincinnati 25
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
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
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Not applicableEnded earlyNCT03810508
What this trial is testing

A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)

Who this might be right for
Charcot-Marie-Tooth DiseaseHereditary Motor and Sensory Neuropathy IV
Neurogene Inc. 21
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Not applicableLooking for participantsNCT01668186
What this trial is testing

Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)

Who this might be right for
Peroxisome Biogenesis DisorderZellweger Spectrum DisorderRCDP - Rhizomelic Chondrodysplasia Punctata+8 more
McGill University Health Centre/Research Institute of the McGill University Health Centre 244