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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableStudy completedNCT03057002
What this trial is testing

UTSW HP [13-C] Pyruvate Injection in HCM

Who this might be right for
Cardiomyopathy, HypertrophicDilated CardiomyopathyDuchenne Muscular Dystrophy+4 more
University of Texas Southwestern Medical Center 7
Not applicableUnknownNCT01613495
What this trial is testing

Ghrelin Suppression by Octreotide in Prader-Willi

Who this might be right for
Prader Willis Syndrome
Oregon Health and Science University 2
Large-scale testing (Phase 3)Ended earlyNCT05764239
What this trial is testing

Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)

Who this might be right for
Phenylketonuria
Synlogic 35
Not applicableUnknownNCT05914727
What this trial is testing

Biomarkers Of Lung InVolvement In ASMD

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 68
Testing effectiveness (Phase 2)Study completedNCT02437253
What this trial is testing

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
Post-approval studies (Phase 4)Looking for participantsNCT04667494
What this trial is testing

Ultrasound Therapy In Cardiac Amyloidosis

Who this might be right for
Amyloidosis Cardiac
Brigham and Women's Hospital 70
Post-approval studies (Phase 4)Looking for participantsNCT06901245
What this trial is testing

Tirzepatide in PWS, HO and GNSO

Who this might be right for
Prader-Willi SyndromeHypothalamic ObesityObesity/Therapy
Grace Kim 36
Testing effectiveness (Phase 2)Study completedNCT00897221
What this trial is testing

A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
ApoPharma 36
Early research (Phase 1)Study completedNCT00151216
What this trial is testing

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 10
Not applicableWithdrawnNCT04055532
What this trial is testing

Biomarkers in Neurodegenerative Diseases

Who this might be right for
Mild Cognitive Impairment (MCI)Alzheimer Disease (AD)Dementia With Lewy Bodies (DLB)+8 more
University of Minnesota
Not applicableLooking for participantsNCT05848271
What this trial is testing

Natural History Study of Patients with HPDL Mutations

Who this might be right for
Mitochondrial EncephalomyopathiesHereditary Spastic ParaplegiaSpastic Paraplegia+4 more
University of California, San Diego 50
Not applicableLooking for participantsNCT07728032
What this trial is testing

Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU

Who this might be right for
Phenylketonuria
Birmingham Women's and Children's NHS Foundation Trust 148
Testing effectiveness (Phase 2)Study completedNCT01907087
What this trial is testing

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Who this might be right for
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+1 more
BioMarin Pharmaceutical 24
Not applicableStudy completedNCT04943393
What this trial is testing

Remote Neurocognitive and Psychological Assessment in PKU

Who this might be right for
Phenylketonurias
University of Missouri-Columbia 84
Not applicableStudy completedNCT00001972
What this trial is testing

PET Scan of Brain Metabolism in Relation to Age and Disease

Who this might be right for
Alzheimer's DiseaseBrain NeoplasmNiemann Pick Disease
National Institute of Neurological Disorders and Stroke (NINDS) 123
Not applicableUnknownNCT06324877
What this trial is testing

Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside

Who this might be right for
Ataxia Telangiectasia
The University of Queensland 10
Not applicableStudy completedNCT06363357
What this trial is testing

The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder

Who this might be right for
Muscular Dystrophy, DuchenneOrthotic DevicesUpper Extremity+6 more
Seoul National University Hospital 8
Not applicableUnknownNCT04991701
What this trial is testing

A National Retrospective Population Based Cohort Study of the Natural History of Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia
University of Nottingham 178
Not applicableNot Yet RecruitingNCT04252001
What this trial is testing

Growing up With the Young Endocrine Support System (YESS!)

Who this might be right for
Congenital Adrenal HyperplasiaHypogonadotropic HypogonadismGrowth Hormone Deficiency+6 more
dr. Laura C. G. de Graaff-Herder 160
Testing effectiveness (Phase 2)Study completedNCT02402309
What this trial is testing

Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)

Who this might be right for
Sjögren-Larsson Syndrome
Aldeyra Therapeutics, Inc. 12
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