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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT00744692
What this trial is testing

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Who this might be right for
Non Malignant DisordersImmunodeficienciesCongenital Marrow Failures+5 more
Duke University 22
Not applicableStudy completedNCT03081832
What this trial is testing

Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.

Who this might be right for
Prader-Willi Syndrome
University Hospital, Toulouse 34
Testing effectiveness (Phase 2)Study completedNCT05485987
What this trial is testing

Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
PTC Therapeutics 5
Not applicableLooking for participantsNCT06096675
What this trial is testing

CAPACITY (Cardiac Amyloidosis and Physical ACtivITY) Study

Who this might be right for
Cardiac Amyloidosis
Wake Forest University Health Sciences 50
Large-scale testing (Phase 3)Active Not RecruitingNCT04348136
What this trial is testing

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 27
Testing effectiveness (Phase 2)Looking for participantsNCT05791864
What this trial is testing

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2
Tern Therapeutics, LLC 16
Not applicableStudy completedNCT05252819
What this trial is testing

Whole Body MRI for Cancer Surveillance in A-T

Who this might be right for
Ataxia Telangiectasia
University of Nottingham 32
Not applicableStudy completedNCT00074932
What this trial is testing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company 9
Not applicableLooking for participantsNCT06121011
What this trial is testing

A Global Prospective Observational Registry of Patients With Pompe Disease

Who this might be right for
Pompe Disease
Amicus Therapeutics 500
Early research (Phase 1)Not Yet RecruitingNCT06735638
What this trial is testing

Outside the Cage (OTC) Robotic Esophagectomy

Who this might be right for
Esophageal Cancer
Centre hospitalier de l'Université de Montréal (CHUM) 31
Not applicableStudy completedNCT02860338
What this trial is testing

COMPARATIVE EFFECTIVENESS OF MCI and DEMENTIA TREATMENTS IN A COMMUNITY-BASED DEMENTIA PRACTICE

Who this might be right for
Mild Cognitive ImpairmentDementiaHypoxia+14 more
Clionsky Neuro Systems Inc. 900
Not applicableLooking for participantsNCT05813678
What this trial is testing

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Who this might be right for
Phenylketonuria (PKU)
BioMarin Pharmaceutical 450
Not applicableStudy completedNCT05051657
What this trial is testing

Evaluation of the Express Plus Range

Who this might be right for
PhenylketonuriasPKUHomocystinuria+3 more
Vitaflo International, Ltd 28
Not applicableLooking for participantsNCT07154381
What this trial is testing

Hybrid Florbetaben PET/MRI for Imaging of Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
University Hospital, Essen 25
Testing effectiveness (Phase 2)Study completedNCT00551200
What this trial is testing

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorders
Amgen 14
Testing effectiveness (Phase 2)Study completedNCT01721733
What this trial is testing

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

Who this might be right for
Leigh Syndrome
PTC Therapeutics 35
Post-approval studies (Phase 4)Not Yet RecruitingNCT07240649
What this trial is testing

Emerging Indications for Hyperbaric Oxygen Treatment

Who this might be right for
Post-COVID-19 ConditionUlcerative ColitisCrohn Disease+38 more
Jay C. Buckey Jr. 100
Testing effectiveness (Phase 2)Ended earlyNCT01389817
What this trial is testing

Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)

Who this might be right for
Leber's Hereditary Optic Neuropathy (LHON)
Medical College of Wisconsin 4
Not applicableLooking for participantsNCT06330350
What this trial is testing

Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling

Who this might be right for
Quality of LifeIchthyosisPalmoplantar Keratoses+7 more
Maastricht University Medical Center 25
Testing effectiveness (Phase 2)Ended earlyNCT04475549
What this trial is testing

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

Who this might be right for
MELAS
Tisento Therapeutics 8
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