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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)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
Not applicableActive Not RecruitingNCT05596760
What this trial is testing

Promoting Goals-of-Care Discussions for Patients With Memory Problems and Their Caregivers

Who this might be right for
DementiaDementia, VascularMixed Dementias+14 more
University of Washington 1,802
Not applicableApproved For MarketingNCT00954460
What this trial is testing

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire
Testing effectiveness (Phase 2)Looking for participantsNCT06488313
What this trial is testing

Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

Who this might be right for
OTC DeficiencyOrnithine Transcarbamylase DeficiencyOTCD
Arcturus Therapeutics, Inc. 9
Not applicableLooking for participantsNCT06178120
What this trial is testing

Disease Progression in Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Minoryx Therapeutics, S.L. 40
Not applicableNot Yet RecruitingNCT07567508
What this trial is testing

Early Detection of Concealed Cardiac Amyloidosis Using AI-ECG and CT-Derived Extracellular Volume in Patients With Atrial Fibrillation

Who this might be right for
Atrial Fibrillation (AF)Cardiac Amyloidosis
Samsung Medical Center 500
Large-scale testing (Phase 3)Study completedNCT00947297
What this trial is testing

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

Who this might be right for
Urea Cycle Disorders
Amgen 60
Not applicableUnknownNCT06279052
What this trial is testing

Adolescent Projections During Transition in Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
University Hospital, Toulouse 20
Large-scale testing (Phase 3)Looking for participantsNCT02000089
What this trial is testing

The Cancer of the Pancreas Screening-5 CAPS5)Study

Who this might be right for
Pancreas CancerPeutz-Jeghers Syndrome (PJS)Gene Mutation+2 more
Johns Hopkins University 9,000
Not applicableUnknownNCT05938218
What this trial is testing

Virtual Reality Assisted Patient Empowerment: Diagnose ATTR-Amyloidosis And Start Treatment

Who this might be right for
Amyloidosis CardiacVirtual Reality
Heinrich-Heine University, Duesseldorf 60
Not applicableEnded earlyNCT05229549
What this trial is testing

Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria

Who this might be right for
Phenylketonurias
APR Applied Pharma Research s.a. 5
Not applicableUnknownNCT04847713
What this trial is testing

Early Detection and Follow-Up of Patients With Fabry's Disease

Who this might be right for
Fabry's Disease
Hospital Clinico Universitario de Santiago 100
Not applicableStudy completedNCT00474643
What this trial is testing

Correlation of Hyperghrelinemia With Carotid Artery Intima-Media Thickness in Children With Prader-Willi Syndrome

Who this might be right for
Prader Willi SyndromeObesity
Samsung Medical Center 51
Not applicableStudy completedNCT00001813
What this trial is testing

Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy

Who this might be right for
Cockayne SyndromeSkin NeoplasmsXeroderma Pigmentosum+2 more
National Cancer Institute (NCI) 709
Not applicableLooking for participantsNCT04463316
What this trial is testing

GROWing Up With Rare GENEtic Syndromes

Who this might be right for
Prader-Willi SyndromePWS-like SyndromeSilver Russel Syndrome+30 more
dr. Laura C. G. de Graaff-Herder 600
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07641140
What this trial is testing

Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease

Who this might be right for
Wilson's Disease
Lingyi Biotech Co., Ltd. 18
Testing effectiveness (Phase 2)WithdrawnNCT03831425
What this trial is testing

Mitochondrial Complex I Dysfunction in PWS

Who this might be right for
Prader-Willi Syndrome
The Hospital for Sick Children
Not applicableActive Not RecruitingNCT04406818
What this trial is testing

Metabolic and Hemodynamic Reserve in Pediatric SCA

Who this might be right for
Child, OnlyBrain DiseasesSickle Cell Disease+1 more
Washington University School of Medicine 120
Not applicableLooking for participantsNCT05938543
What this trial is testing

Cerebellar TMS and Satiety in Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
Brigham and Women's Hospital 20
Not applicableUnknownNCT05413876
What this trial is testing

Fabry Exercise Intolerance Study

Who this might be right for
Fabry DiseaseFabry Disease, Cardiac Variant
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 30
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