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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Not applicableStudy completedNCT03626584
What this trial is testing

PET/MR Imaging In Patients With Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
University of Edinburgh 33
Large-scale testing (Phase 3)Ended earlyNCT00712426
What this trial is testing

Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)

Who this might be right for
Huntington's Disease
Massachusetts General Hospital 553
Early research (Phase 1)Study completedNCT00258778
What this trial is testing

Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)

Who this might be right for
Gaucher Disease
Protalix 6
Early research (Phase 1)Ended earlyNCT00004058
What this trial is testing

12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder

Who this might be right for
Chronic Myeloproliferative DisordersLeukemiaLymphoma+4 more
University of Medicine and Dentistry of New Jersey
Testing effectiveness (Phase 2)UnknownNCT01773278
What this trial is testing

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

Who this might be right for
Smith-Lemli-Opitz SyndromeCone-Rod DystrophyHearing Loss
University of Colorado, Denver 100
Not applicableLooking for participantsNCT04806620
What this trial is testing

Unhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation Research

Who this might be right for
Post-Acute COVID-19 SyndromeME/CFSRheumatic Arthritis+41 more
Brain Inflammation Collaborative 10,000
Not applicableLooking for participantsNCT05484570
What this trial is testing

Natural History Study for DNA Repair Disorders

Who this might be right for
DNA Repair DisorderCockayne SyndromeXeroderma Pigmentosum+1 more
University of Minnesota 40
Not applicableUnknownNCT03786497
What this trial is testing

Protecting Brains and Saving Futures - the PBSF Protocol

Who this might be right for
Brain InjuriesCongenital Heart DiseaseNewborn Morbidity+14 more
Protecting Brains Saving Futures 2,268
Not applicableUnknownNCT06367842
What this trial is testing

Orthopaedic Specimen Pathology and Early Diagnosis of ATTR Cardiopathy (ATTR-ORTHO)

Who this might be right for
ATTR AmyloidosisCardiopathyCarpal Tunnel Syndrome+3 more
Institut Mutualiste Montsouris 150
Testing effectiveness (Phase 2)Study completedNCT01038570
What this trial is testing

Comparative Study Between Prader-Willi Patients Who Take Oxytocin Versus Placebo

Who this might be right for
Prader Willi Syndrome
University Hospital, Toulouse 24
Not applicableLooking for participantsNCT06523582
What this trial is testing

Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients

Who this might be right for
Neuroendocrine NeoplasmNeuroendocrine Neoplasm of Gastrointestinal TractNeuroendocrine Neoplasm of Lung+23 more
Universidad Nacional Autonoma de Mexico 750
Testing effectiveness (Phase 2)WithdrawnNCT03767270
What this trial is testing

Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency
Translate Bio, Inc.
Not applicableStudy completedNCT01565005
What this trial is testing

Microcephaly Genetic Deficiency in Neural Progenitors

Who this might be right for
Microcephaly
Assistance Publique - Hôpitaux de Paris 98
Post-approval studies (Phase 4)Study completedNCT04718779
What this trial is testing

Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Who this might be right for
Gaucher Disease
Takeda 4
Large-scale testing (Phase 3)Study completedNCT01939561
What this trial is testing

Lamotrigine as Treatment of Myotonia

Who this might be right for
Dystrophia Myotonica Type 1Myotonia CongenitaParamyotonia Congenita+2 more
Grete Andersen, MD 27
Not applicableStudy completedNCT04862273
What this trial is testing

CMR T1 Mapping for Diagnosis of Cardiac Amyloidosis

Who this might be right for
Heart Failure NYHA Class IIHeart Failure NYHA Class IIIHeart Failure NYHA Class IV+4 more
University of Leipzig 112
Large-scale testing (Phase 3)Study completedNCT01298141
What this trial is testing

Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

Who this might be right for
Fabry Disease
Shire 171
Not applicableLooking for participantsNCT00912041
What this trial is testing

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

Who this might be right for
TetraplegiaSpinal Cord InjuriesAmyotrophic Lateral Sclerosis+3 more
Leigh R. Hochberg, MD, PhD. 27
Post-approval studies (Phase 4)Study completedNCT00081497
What this trial is testing

The Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 67
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