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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Looking for participantsNCT07465718
What this trial is testing

Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

Who this might be right for
Wilson's Disease
Orphalan 38
Not applicableStudy completedNCT03120013
What this trial is testing

Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia

Who this might be right for
Ataxia, CerebellarCerebellar AtaxiaSpinocerebellar Ataxias+8 more
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia 21
Large-scale testing (Phase 3)Ended earlyNCT00122980
What this trial is testing

Stroke With Transfusions Changing to Hydroxyurea

Who this might be right for
HemochromatosisCerebrovascular AccidentAnemia, Sickle Cell+1 more
St. Jude Children's Research Hospital 134
Not applicableStudy completedNCT00006142
What this trial is testing

Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
National Center for Research Resources (NCRR)
Not applicableStudy completedNCT05557162
What this trial is testing

Artificial Intelligence Enhanced ECG to Detect Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
Mayo Clinic 20,143
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi ETS 20
Testing effectiveness (Phase 2)Study completedNCT01942590
What this trial is testing

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

Who this might be right for
Pompe Disease
Dwight Koeberl, M.D., Ph.D. 17
Early research (Phase 1)Study completedNCT01747980
What this trial is testing

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Protalix 16
Not applicableStudy completedNCT05409846
What this trial is testing

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

Who this might be right for
Fabry Disease
Universidade do Porto 409
Not applicableUnknownNCT02605603
What this trial is testing

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Who this might be right for
Gaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 30
Not applicableNot Yet RecruitingNCT05017402
What this trial is testing

Higher Dose of Alglucosidase Alpha for Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 36
Not applicableLooking for participantsNCT00567073
What this trial is testing

Pompe Pregnancy Sub-Registry

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 20
Testing effectiveness (Phase 2)Study completedNCT05758922
What this trial is testing

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Disease, Type C
Azafaros B.V. 13
Large-scale testing (Phase 3)Study completedNCT01347073
What this trial is testing

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

Who this might be right for
Urea Cycle Disorders
Amgen 23
Not applicableUnknownNCT02527239
What this trial is testing

Prevalence of Pompe's Disease in Respiratory Clinics

Who this might be right for
Pompe's Disease
Royal Brompton & Harefield NHS Foundation Trust 400
Large-scale testing (Phase 3)Active Not RecruitingNCT04849741
What this trial is testing

Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

Who this might be right for
Alexander Disease
Ionis Pharmaceuticals, Inc. 54
Large-scale testing (Phase 3)Not Yet RecruitingNCT06391645
What this trial is testing

Nerve Growth Factor Encapsulated With 2-methacryloyloxyethyl Phosphorylcholine Nanocapsules in the Treatment of Amyotrophic Lateral Sclerosis

Who this might be right for
Amyotrophic Lateral Sclerosis
Beijing Tiantan Hospital 60
Testing effectiveness (Phase 2)Study completedNCT02140151
What this trial is testing

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

Who this might be right for
Age-related Macular Degeneration
Southern California Desert Retina Consultants, MC 108
Not applicableStudy completedNCT00309400
What this trial is testing

The Early History of Universal Screening for Metabolic Disorders

Who this might be right for
PhenylketonuriaGalactosemiaInborn Errors of Metabolism
University of Miami 10
Testing effectiveness (Phase 2)Study completedNCT00053573
What this trial is testing

RhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 20
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