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Results for “Hereditary Muscular Dystrophy”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT02851797
What this trial is testing

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Italfarmaco 179
Not applicableEnded earlyNCT03867435
What this trial is testing

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

Who this might be right for
Myotonic Dystrophy Type-1Myotonic Dystrophy Type-2
National Institute of Neurological Disorders and Stroke (NINDS) 4
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Testing effectiveness (Phase 2)Study completedNCT05670730
What this trial is testing

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

Who this might be right for
Duchenne Muscular DystrophyExon 44
Avidity Biosciences, Inc. 70
Not applicableEnrolling By InvitationNCT06270719
What this trial is testing

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 500
Not applicableUnknownNCT06109103
What this trial is testing

Telerehabilitation-based Motor Imagery Training in Children With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Hacettepe University 36
Large-scale testing (Phase 3)Active Not RecruitingNCT05693142
What this trial is testing

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
REGENXBIO Inc. 65
Not applicableStudy completedNCT05209087
What this trial is testing

Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Hacettepe University 30
Not applicableNot Yet RecruitingNCT07101185
What this trial is testing

Transition to Adulthood in People With Muscular Dystrophy

Who this might be right for
Duchenne / Becker Muscular Dystrophy
IRCCS Eugenio Medea 250
Not applicableStudy completedNCT03981289
What this trial is testing

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

Who this might be right for
Limb Girdle Muscular DystrophyMuscular Dystrophies
Virginia Commonwealth University 116
Post-approval studies (Phase 4)Not Yet RecruitingNCT07542314
What this trial is testing

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 20
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
Not applicableLooking for participantsNCT06708468
What this trial is testing

Personalized Training for People With Rare Neuromuscular Disorders

Who this might be right for
Neuromuscular Diseases (NMD)Charcot Marie Tooth Disease (CMT)Facioscapulohumeral Muscular Dystrophy+1 more
Oslo University Hospital 120
Not applicableEnrolling By InvitationNCT06101940
What this trial is testing

Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)

Who this might be right for
Myotonic Dystrophy 1
Huashan Hospital 1,000
Testing effectiveness (Phase 2)Ended earlyNCT02907619
What this trial is testing

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Pfizer 59
Large-scale testing (Phase 3)Ended earlyNCT01803412
What this trial is testing

The Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

Who this might be right for
Muscular Dystrophies
BioMarin Pharmaceutical 53
Early research (Phase 1)Study completedNCT03508947
What this trial is testing

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Wave Life Sciences USA, Inc. 36
Large-scale testing (Phase 3)Study completedNCT04624750
What this trial is testing

Open Label Study in Adolescents and Children With Myotonic Disorders

Who this might be right for
Myotonic Dystrophy
Lupin Ltd. 12
Not applicableLooking for participantsNCT05394506
What this trial is testing

Modifying Factors in Striated Muscle Laminopathies

Who this might be right for
LaminopathiesEmery Dreifuss Muscular Dystrophy 2LMNA-Related Congenital Muscular Dystrophy+1 more
Institut National de la Santé Et de la Recherche Médicale, France 40
Testing effectiveness (Phase 2)Study completedNCT03642860
What this trial is testing

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Who this might be right for
Tarui DiseaseDebrancher DeficiencyGYG1 DEFICIENCY
Rigshospitalet, Denmark 3
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