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Results for “X-linked Genetic Diseases”

1,800 trials

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Showing 20 of 1,800 results

Not applicableUnknownNCT05981313
What this trial is testing

Viscoelastic Properties of Lower Extremity Muscles in Patients With Hemophilia

Who this might be right for
HemophiliaHemophilia AHemophilia B
Hasan Kalyoncu University 40
Not applicableNot Yet RecruitingNCT03645486
What this trial is testing

Lentiviral Gene Therapy for CGD

Who this might be right for
Chronic Granulomatous Disease
Shenzhen Geno-Immune Medical Institute 10
Large-scale testing (Phase 3)Ended earlyNCT02090959
What this trial is testing

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 219
Post-approval studies (Phase 4)Study completedNCT01070511
What this trial is testing

Tadalafil in Becker Muscular Dystrophy

Who this might be right for
Becker Muscular Dystrophy
Cedars-Sinai Medical Center 48
Not applicableUnknownNCT02843334
What this trial is testing

Study of the Prevalence of Fabry Disease in French Dialysis Patients

Who this might be right for
Fabry DiseaseEnd Stage Renal DiseaseRenal Dialysis
Hospices Civils de Lyon 6,000
Large-scale testing (Phase 3)Study completedNCT00925301
What this trial is testing

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 67
Testing effectiveness (Phase 2)Study completedNCT00071877
What this trial is testing

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableStudy completedNCT05330195
What this trial is testing

Bicycle Ergometer Training in Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Hacettepe University 23
Not applicableWithdrawnNCT07607600
What this trial is testing

Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia

Who this might be right for
X-linked Hypophosphatemia (XLH)
Novo Nordisk A/S
Testing effectiveness (Phase 2)Study completedNCT03507686
What this trial is testing

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111

Who this might be right for
Choroideremia
Biogen 66
Not applicableStudy completedNCT03314207
What this trial is testing

Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

Who this might be right for
X-Linked Retinitis Pigmentosa
Beacon Therapeutics 14
Testing effectiveness (Phase 2)Study completedNCT01357239
What this trial is testing

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Who this might be right for
Fragile X Syndrome
Novartis Pharmaceuticals 139
Large-scale testing (Phase 3)Ended earlyNCT04988867
What this trial is testing

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Who this might be right for
Rett Syndrome
ACADIA Pharmaceuticals Inc. 15
Testing effectiveness (Phase 2)Looking for participantsNCT06138639
What this trial is testing

SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Who this might be right for
Duchenne Muscular Dystrophy
Solid Biosciences Inc. 60
Large-scale testing (Phase 3)Study completedNCT01254019
What this trial is testing

A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophies
GlaxoSmithKline 186
Not applicableStudy completedNCT03596554
What this trial is testing

X-linked Hypophosphatemia and FGF21

Who this might be right for
X-linked Hypophosphatemia
Hospices Civils de Lyon 39
Not applicableStudy completedNCT02952482
What this trial is testing

Newborn Screening for Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
National Taiwan University Hospital 45,796
Not applicableUnknownNCT06378203
What this trial is testing

Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]

Who this might be right for
Muscular DystrophiesLimb Girdle Muscular DystrophyFacio-Scapulo-Humeral Dystrophy+1 more
IRCCS Eugenio Medea 10
Not applicableStudy completedNCT05464446
What this trial is testing

Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular DystrophyLower Urinary Tract SymptomsMuscle Weakness+4 more
Lokman Hekim University 45
Large-scale testing (Phase 3)Active Not RecruitingNCT05367960
What this trial is testing

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Who this might be right for
Fragile X Syndrome
Tetra Discovery Partners 314
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