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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349
What this trial is testing

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more
Spark Therapeutics, Inc. 4
Not applicableStudy completedNCT00937794
What this trial is testing

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Who this might be right for
Hunter Syndrome
Shire 33
Testing effectiveness (Phase 2)Looking for participantsNCT07303296
What this trial is testing

Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

Who this might be right for
Leber Hereditary Optic Disease
GenSight Biologics 14
Not applicableStudy completedNCT04224142
What this trial is testing

Evaluation of PKU Sphere in Maternal PKU

Who this might be right for
Maternal Phenylketonuria
Vitaflo International, Ltd 12
Not applicableWithdrawnNCT01459302
What this trial is testing

Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders

Who this might be right for
Amyotrophic Lateral SclerosisFrontotemporal DementiaPLS+7 more
University of Massachusetts, Worcester
Not applicableLooking for participantsNCT07277361
What this trial is testing

Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment

Who this might be right for
Fabry DiseaseAged 65 Years or OlderAlpha Galactosidase A Deficiency+1 more
Wladimir MAUHIN, Dr 100
Testing effectiveness (Phase 2)UnknownNCT05447494
What this trial is testing

Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
CANbridge (Suzhou) Bio-pharma Co., Ltd. 40
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Early research (Phase 1)WithdrawnNCT00000856
What this trial is testing

A Phase I/II Pilot Treatment Study Of CSF Penetration And Response To Ganciclovir And Foscarnet In CMV Neurologic Disease.

Who this might be right for
EncephalopathyHIV InfectionsRadiculitis
National Institute of Allergy and Infectious Diseases (NIAID)
Not applicableStudy completedNCT01663896
What this trial is testing

Observational Study of OCT in a Patients Undergoing FFR

Who this might be right for
Coronary Artery Disease
Abbott Medical Devices 418
Early research (Phase 1)Study completedNCT02082327
What this trial is testing

Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers

Who this might be right for
Fabry Disease
Amicus Therapeutics 31
Early research (Phase 1)Study completedNCT00418561
What this trial is testing

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 13
Not applicableStudy completedNCT02956954
What this trial is testing

Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease

Who this might be right for
Anderson-Fabry Disease
University Hospital, Rouen 26
Not applicableUnknownNCT03683966
What this trial is testing

MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study

Who this might be right for
Rare DiseasesFabry DiseaseAdherence, Medication+1 more
Wuerzburg University Hospital 30
Not applicableWithdrawnNCT03145779
What this trial is testing

Evaluation of Phenotypic Variability in Fabry Disease

Who this might be right for
Fabry Disease
Boston Children's Hospital
Not applicableEnrolling By InvitationNCT05697861
What this trial is testing

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Who this might be right for
Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyTransthyretin-Related (ATTR) Familial Amyloid CardiomyopathyWild-Type Transthyretin Cardiac Amyloidosis
Intellia Therapeutics 72
Not applicableUnknownNCT03602378
What this trial is testing

QoL and Stress in Parents of Children With Developmental Disabilities and Chronic Disease

Who this might be right for
Developmental DisabilityChronic DiseaseDown Syndrome+7 more
University of Split, School of Medicine 592
Large-scale testing (Phase 3)Study completedNCT01124643
What this trial is testing

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

Who this might be right for
Fabry Disease
Shire 35
Large-scale testing (Phase 3)Active Not RecruitingNCT06031259
What this trial is testing

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Who this might be right for
Hunter Syndrome
Takeda 6
Very early researchStudy completedNCT00640003
What this trial is testing

Baclofen Treatment of Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia
Johns Hopkins University 10
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