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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT06795152
What this trial is testing

Rare Glycogen Storage Diseases Natural History Study

Who this might be right for
Glycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more
Duke University 200
Large-scale testing (Phase 3)Study completedNCT00212355
What this trial is testing

Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.

Who this might be right for
Wilson's Disease
Nobelpharma 37
Not applicableActive Not RecruitingNCT05943678
What this trial is testing

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.

Who this might be right for
Pompe DiseaseMcArdle Disease
Medical University of Vienna 30
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Very early researchLooking for participantsNCT06272149
What this trial is testing

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Who this might be right for
Type II Gaucher Disease
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 6
Not applicableNot Yet RecruitingNCT07313085
What this trial is testing

Mechanisms of Fecal Incontinence in Wolfram Syndrome

Who this might be right for
Wolfram SyndromeFecal Incontinence
Hôpital Necker-Enfants Malades 20
Not applicableLooking for participantsNCT07575347
What this trial is testing

Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)

Who this might be right for
Periodontal DiseasePeriodontitisCKD+6 more
Stefan Lujinschi 100
Early research (Phase 1)Looking for participantsNCT05302271
What this trial is testing

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Who this might be right for
Friedreich AtaxiaCardiomyopathiesCardiac Hypertrophy+1 more
Weill Medical College of Cornell University 25
Testing effectiveness (Phase 2)Study completedNCT02912793
What this trial is testing

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 12
Large-scale testing (Phase 3)Study completedNCT04138277
What this trial is testing

Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 119
Not applicableActive Not RecruitingNCT00787865
What this trial is testing

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Who this might be right for
Krabbe Disease
University of Pittsburgh 100
Testing effectiveness (Phase 2)Study completedNCT02404337
What this trial is testing

Betaine METABOLISM OF PATIENTS With Homocystinuria

Who this might be right for
Homocystinuria
Assistance Publique - Hôpitaux de Paris 12
Not applicableStudy completedNCT04879277
What this trial is testing

Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
University Hospital, Tours 40
Testing effectiveness (Phase 2)Study completedNCT00742664
What this trial is testing

Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project

Who this might be right for
Prader Willi SyndromeObsessive-compulsive Disorder
University of South Florida 24
Not applicableStudy completedNCT03114371
What this trial is testing

Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years

Who this might be right for
Prader-Willi Syndrome
University Hospital, Toulouse 40
Not applicableStudy completedNCT04239521
What this trial is testing

The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata

Who this might be right for
Alopecia AreataDepressive EpisodeRecurrent Depressive Disorder+33 more
Momentum Data 51,955
Not applicableLooking for participantsNCT07013292
What this trial is testing

Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Centre Hospitalier Universitaire de Nice 40
Testing effectiveness (Phase 2)Study completedNCT00229632
What this trial is testing

Idebenone to Treat Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
National Institute of Neurological Disorders and Stroke (NINDS) 51
Not applicableActive Not RecruitingNCT04875416
What this trial is testing

Phenotype, Genotype and Biomarkers 2

Who this might be right for
Amyotrophic Lateral SclerosisHereditary Spastic ParaplegiaPrimary Lateral Sclerosis+2 more
University of Miami 217
Large-scale testing (Phase 3)Study completedNCT00712348
What this trial is testing

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Who this might be right for
Gaucher Disease
Pfizer 31
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