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Results for “X-linked Genetic Diseases”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT03058185
What this trial is testing

Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)

Who this might be right for
LaminopathiesEmerinopathies
Pitié-Salpêtrière Hospital 800
Testing effectiveness (Phase 2)Study completedNCT02715115
What this trial is testing

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Who this might be right for
Rett Syndrome
Neuren Pharmaceuticals Limited 82
Not applicableStudy completedNCT03054389
What this trial is testing

Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews

Who this might be right for
Hemophilia B
Baxalta now part of Shire 27
Not applicableUnknownNCT04014985
What this trial is testing

Patients With RETT Syndrome

Who this might be right for
Rett Syndrome
Assistance Publique Hopitaux De Marseille 100
Large-scale testing (Phase 3)Study completedNCT03569891
What this trial is testing

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Who this might be right for
Hemophilia B
CSL Behring 67
Not applicableStudy completedNCT03529240
What this trial is testing

Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption

Who this might be right for
PerformanceEnergyGait+1 more
Hacettepe University 45
Not applicableStudy completedNCT04750785
What this trial is testing

Assess Choroideremia (CHM) Health Outcomes

Who this might be right for
Choroideremia
Biogen 46
Testing effectiveness (Phase 2)Ended earlyNCT01168908
What this trial is testing

Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular Dystrophy
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 20
Testing effectiveness (Phase 2)Study completedNCT00990691
What this trial is testing

Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome

Who this might be right for
Rett Syndrome
Assistance Publique Hopitaux De Marseille 36
Not applicableStudy completedNCT06379906
What this trial is testing

Upper Extremity Muscle Strength, Balance and Functional Skills in DMD

Who this might be right for
Duchenne Muscular Dystrophy
Halic University 32
Not applicableLooking for participantsNCT07286565
What this trial is testing

Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening

Who this might be right for
Spinal Muscular Atrophy (SMA)Duchenne Muscular Dystrophy (DMD)
Centre Hospitalier Universitaire de Liege 100
Testing effectiveness (Phase 2)Study completedNCT02489344
What this trial is testing

Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 8
Early research (Phase 1)Study completedNCT02376816
What this trial is testing

Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Jerry R. Mendell 2
Early research (Phase 1)Study completedNCT06475404
What this trial is testing

The Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 18
Not applicableStudy completedNCT03582449
What this trial is testing

Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Who this might be right for
Hunter Syndrome
Shire 5
Testing effectiveness (Phase 2)Study completedNCT01894958
What this trial is testing

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

Who this might be right for
Fragile X Syndrome
Neuren Pharmaceuticals Limited 72
Not applicableLooking for participantsNCT06560242
What this trial is testing

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant

Who this might be right for
Fragile X Syndrome
Children's Hospital Medical Center, Cincinnati 30
Testing effectiveness (Phase 2)Looking for participantsNCT07038824
What this trial is testing

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
Entrada Therapeutics, Inc. 24
Not applicableLooking for participantsNCT06227780
What this trial is testing

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

Who this might be right for
Fragile X SyndromeAutism Spectrum DisorderAutistic Disorder+1 more
Children's Hospital Medical Center, Cincinnati 180
Not applicableStudy completedNCT03848442
What this trial is testing

'Uptime' Participation Intervention in Girls and Women With Rett Syndrome

Who this might be right for
Rett Syndrome
Rigshospitalet, Denmark 14
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