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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00975689
What this trial is testing

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

Who this might be right for
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 35
Testing effectiveness (Phase 2)Study completedNCT00358150
What this trial is testing

The Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Who this might be right for
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 26
Not applicableStudy completedNCT01549015
What this trial is testing

Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle Function

Who this might be right for
Urea Cycle Disorders
Cytonet GmbH & Co. KG 37
Testing effectiveness (Phase 2)Study completedNCT06792240
What this trial is testing

Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study

Who this might be right for
Phenylketonuria (PKU)
ASST Santi Paolo e Carlo 84
Large-scale testing (Phase 3)Study completedNCT00332189
What this trial is testing

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 111
Testing effectiveness (Phase 2)Ended earlyNCT01977820
What this trial is testing

Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 2
Post-approval studies (Phase 4)Looking for participantsNCT07695701
What this trial is testing

Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis

Who this might be right for
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)AmyloidosisAmyloid Cardiomyopathy+1 more
Eidos Therapeutics, a BridgeBio company 150
Testing effectiveness (Phase 2)Active Not RecruitingNCT04537377
What this trial is testing

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

Who this might be right for
Wilson's Disease
Vivet Therapeutics SAS 4
Testing effectiveness (Phase 2)Study completedNCT03950050
What this trial is testing

Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 40
Not applicableLooking for participantsNCT06865482
What this trial is testing

Clinical Course Of Disease In Participants With FA-CM

Who this might be right for
Friedreich AtaxiaCardiomyopathy
Lexeo Therapeutics 65
Not applicableEnded earlyNCT04073888
What this trial is testing

Study of the Spermatic Characteristics of Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Hospital, Bordeaux 22
Not applicableLooking for participantsNCT04885179
What this trial is testing

SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)

Who this might be right for
SphingolipidosesEnzyme Deficiency
University of California, San Francisco 120
Not applicableUnknownNCT04319796
What this trial is testing

European Registry on Rare Neurological Diseases

Who this might be right for
Rare Diseases
University Hospital Tuebingen 5,000
Not applicableStudy completedNCT01355146
What this trial is testing

Home Therapy With Replagal in Fabry Disease

Who this might be right for
Fabry Disease
Shire 127
Not applicableStudy completedNCT05412160
What this trial is testing

Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD

Who this might be right for
COPDDyspneaArgininemia
University of Milan 153
Very early researchUnknownNCT03201627
What this trial is testing

Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

Who this might be right for
Niemann-Pick Disease, Type C1
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 18
Post-approval studies (Phase 4)Study completedNCT01132690
What this trial is testing

A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer 11
Not applicableStudy completedNCT05497050
What this trial is testing

The Effect of Nursing Empowerment Program

Who this might be right for
Phenylketonurias
Cukurova University 36
Not applicableStudy completedNCT01891422
What this trial is testing

Longitudinal Studies of the Glycoproteinoses

Who this might be right for
AspartylglucosaminuriaFucosidosisGalactosialidosis+6 more
Greenwood Genetic Center 100
Not applicableActive Not RecruitingNCT04564495
What this trial is testing

Home Based Tele-exercise for People With Chronic Neurological Impairments

Who this might be right for
Neurologic DisorderStrokeSpinal Cord Injuries+8 more
Burke Medical Research Institute 60
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