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Results for “X-linked Genetic Diseases”

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Showing 20 of 0 results

Early research (Phase 1)Looking for participantsNCT05709288
What this trial is testing

Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old

Who this might be right for
Hemophilia B
Institute of Hematology & Blood Diseases Hospital, China 9
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Not applicableEnded earlyNCT02994368
What this trial is testing

"Natural History" Study of Choroideremia

Who this might be right for
Choroideremia
4D Molecular Therapeutics 57
Not applicableStudy completedNCT01165060
What this trial is testing

The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-linked AdrenoleukodystrophyAdrenomyeloneuropathy
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 10
Not applicableStudy completedNCT01506518
What this trial is testing

Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
University of North Carolina, Chapel Hill 60
Not applicableUnknownNCT03372655
What this trial is testing

Prognostic Factors Affecting Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Assiut University 82
Testing effectiveness (Phase 2)UnknownNCT05710367
What this trial is testing

Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients

Who this might be right for
Fabry Disease
Albina Nowak, MD 46
Testing effectiveness (Phase 2)Study completedNCT02061137
What this trial is testing

Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome

Who this might be right for
Rett's Syndrome
University Hospital, Basel, Switzerland 6
Not applicableStudy completedNCT02255981
What this trial is testing

Efficacy of Acupuncture in Macular Diseases

Who this might be right for
Age-related Macular DegenerationStargardt DiseaseMacular Dystrophy
Escuela Neijing 33
Not applicableStudy completedNCT05099107
What this trial is testing

Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment

Who this might be right for
Congenital MyopathyNeuromuscular DiseasesMusculoskeletal Diseases+3 more
Vastra Gotaland Region 18
Large-scale testing (Phase 3)Study completedNCT00093171
What this trial is testing

Study Evaluating rFIX; BeneFIX® in Hemophilia B

Who this might be right for
Hemophilia B
Wyeth is now a wholly owned subsidiary of Pfizer 23
Not applicableActive Not RecruitingNCT04946409
What this trial is testing

Burden of Disease and Functional Impairment in XLH

Who this might be right for
X Linked Hypophosphatemia
Wuerzburg University Hospital 60
Not applicableLooking for participantsNCT06147414
What this trial is testing

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Who this might be right for
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, IncludingSickle Cell DiseaseCystic Fibrosis+12 more
Assistance Publique - Hôpitaux de Paris 550
Large-scale testing (Phase 3)Study completedNCT01884311
What this trial is testing

Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases

Who this might be right for
Primary Immune Deficiency DisordersCommon Variable ImmunodeficiencyX-linked Agammaglobulinaemia+1 more
Bio Products Laboratory 38
Testing effectiveness (Phase 2)Ended earlyNCT04004065
What this trial is testing

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 62
Not applicableStudy completedNCT00927992
What this trial is testing

Study Evaluating Liver Transplantation in Haemophilia Patients in Spain

Who this might be right for
HemophiliaHemophilia B
Wyeth is now a wholly owned subsidiary of Pfizer 11
Not applicableUnknownNCT02859363
What this trial is testing

Fabry Disease in Cerebrovascular Disease

Who this might be right for
Fabry Disease
Chang Gung Memorial Hospital 700
Testing effectiveness (Phase 2)Study completedNCT00214500
What this trial is testing

AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 9
Not applicableStudy completedNCT05106764
What this trial is testing

Early Detection of Fabry Disease

Who this might be right for
Fabry Disease
Takeda 50
Large-scale testing (Phase 3)Study completedNCT04587908
What this trial is testing

TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

Who this might be right for
Duchenne Muscular Dystrophy
Taiho Pharmaceutical Co., Ltd. 104
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