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Results for “Genetic Disease, X-Linked”

1,800 trials

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Showing 20 of 1,800 results

Not applicableStudy completedNCT04959188
What this trial is testing

Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)

Who this might be right for
Dyskeratosis CongenitaTelomere Biology Disorders
National Cancer Institute (NCI) 53
Not applicableStudy completedNCT01194141
What this trial is testing

Exercise Training in Barth Syndrome

Who this might be right for
Barth Syndrome
Washington University School of Medicine 4
Not applicableStudy completedNCT03624556
What this trial is testing

Pediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS)

Who this might be right for
Down SyndromeFragile X Syndrome
Parc de Salut Mar 76
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07773246
What this trial is testing

Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD

Who this might be right for
Urea Cycle Disorders, InbornUrea Cycle DisordersOrnithine Transcarbamylase (OTC) Deficiency+8 more
Korro Bio, Inc. 30
Not applicableActive Not RecruitingNCT01633866
What this trial is testing

Pediatric Radio Frequency Coils Generic

Who this might be right for
Duchenne Muscular DystrophyMusculoskeletal Abnormalities
Children's Hospital Medical Center, Cincinnati 75
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableStudy completedNCT00004773
What this trial is testing

Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome

Who this might be right for
Rett Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 95
Not applicableUnknownNCT03198195
What this trial is testing

Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

Who this might be right for
Wiskott-Aldrich Syndrome
Capital Research Institute of Pediatrics 5
Not applicableStudy completedNCT05688072
What this trial is testing

Trunk Oriented Exercises Versus Whole-body Vibration for Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne Type
Cairo University 30
Not applicableStudy completedNCT02740413
What this trial is testing

Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study

Who this might be right for
Hemophilia AHemophilia B
Pfizer 86
Testing effectiveness (Phase 2)Study completedNCT02740972
What this trial is testing

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
NS Pharma, Inc. 16
Large-scale testing (Phase 3)Study completedNCT03417102
What this trial is testing

Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Who this might be right for
Hemophilia AHemophilia B
Genzyme, a Sanofi Company 60
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
Not applicableAvailableNCT06731036
What this trial is testing

Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts

Who this might be right for
Severe Combined ImmunodeficiencyFanconi AnemiaDyskeratosis Congenita+1 more
University of Florida
Testing effectiveness (Phase 2)Study completedNCT01462292
What this trial is testing

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Muscular Dystrophies
GlaxoSmithKline 51
Not applicableEnrolling By InvitationNCT06285071
What this trial is testing

Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors

Who this might be right for
Haemophilia AHaemophilia B
Novo Nordisk A/S 23
Not applicableStudy completedNCT05549999
What this trial is testing

Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment

Who this might be right for
Duchenne Muscular DystrophyAmbulation DifficultyAmbulation Disorder, Neurologic
Hacettepe University 86
Post-approval studies (Phase 4)Study completedNCT01999075
What this trial is testing

Stacking Exercises Aid the Decline in FVC and Sick Time

Who this might be right for
Duchenne Muscular Dystrophy
Children's Hospital of Eastern Ontario 70
Not applicableStudy completedNCT01451281
What this trial is testing

Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

Who this might be right for
Muscular DystrophyMuscular Disease
National Institute of Neurological Disorders and Stroke (NINDS) 35
Not applicableUnknownNCT01646411
What this trial is testing

300 Antibody Diagnostic Test Kit

Who this might be right for
Acute Bacterial InfectionsAcute Viral Infections
The Plasma Source 300
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