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Results for “Congenital Myopathies”

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Enrolling by Invitation

Showing 20 of 0 results

Not applicableLooking for participantsNCT04003363
What this trial is testing

The United Kingdom National Registry for Myotonic Dystrophy

Who this might be right for
Myotonic Dystrophy
Newcastle University 900
Not applicableStudy completedNCT01098708
What this trial is testing

This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Washington University School of Medicine 50
Not applicableActive Not RecruitingNCT05206617
What this trial is testing

3 Year Follow up on ANO5 Patients

Who this might be right for
Neuromuscular DiseasesLimb Girdle Muscular Dystrophy
Rigshospitalet, Denmark 17
Not applicableWithdrawnNCT01457443
What this trial is testing

Biomarker for Pompe Disease (BioPompe)

Who this might be right for
Cardiac DiseasesMuscular WeaknessHepato-splenomegaly+4 more
CENTOGENE GmbH Rostock
Large-scale testing (Phase 3)Active Not RecruitingNCT05881408
What this trial is testing

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 148
Testing effectiveness (Phase 2)WithdrawnNCT04226924
What this trial is testing

Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose

Who this might be right for
Oculopharyngeal Muscular Dystrophy
Bioblast Pharma Ltd.
Not applicableUnknownNCT03998540
What this trial is testing

Improvement of DIAgnostic and Phenotype-genotype Correlation Studies in Patients With MYOpathy Suspected of TITinopathy

Who this might be right for
MyopathyPhenotypic AbnormalityGenetic Disease
University Hospital, Montpellier 50
Not applicableLooking for participantsNCT07194174
What this trial is testing

Effect of Physical Training in Individuals With Hypokalemic and Hyperkalemic Periodic Paralysis

Who this might be right for
Hypokalemic Periodic ParalysisHyperkalemic Periodic Paralysis
Rigshospitalet, Denmark 36
Post-approval studies (Phase 4)Ended earlyNCT04708314
What this trial is testing

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Rare Disease Research, LLC 2
Large-scale testing (Phase 3)Active Not RecruitingNCT04281485
What this trial is testing

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Pfizer 114
Not applicableStudy completedNCT02745938
What this trial is testing

GDF-15 as a Biomarker for Mitochondrial Disease

Who this might be right for
Mitochondrial DiseaseMetabolic MyopathyMuscular Dystrophy
Rigshospitalet, Denmark 97
Early research (Phase 1)Study completedNCT02081625
What this trial is testing

Exploratory Study of NS-065/NCNP-01 in DMD

Who this might be right for
Duchenne Muscular Dystrophy
National Center of Neurology and Psychiatry, Japan 10
Large-scale testing (Phase 3)Enrolling By InvitationNCT06549400
What this trial is testing

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.

Who this might be right for
Myotonic Dystrophy
Lupin Ltd. 176
Early research (Phase 1)Study completedNCT02383511
What this trial is testing

Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet

Who this might be right for
Muscular Dystrophy, Duchenne
Summit Therapeutics 12
Early research (Phase 1)Study completedNCT01519349
What this trial is testing

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis

Who this might be right for
Becker Muscular DystrophySporadic Inclusion Body Myositis
Nationwide Children's Hospital 15
Large-scale testing (Phase 3)Looking for participantsNCT07486934
What this trial is testing

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Who this might be right for
Myotonic Dystrophy Type 1 (DM1)DM1Myotonic Dystrophy+2 more
Dyne Therapeutics 150
Not applicableStudy completedNCT02168114
What this trial is testing

Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle

Who this might be right for
Duchenne Muscular Dystrophy
University of Florida 17
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
Not applicableStudy completedNCT03851107
What this trial is testing

The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities

Who this might be right for
Cerebral PalsySpina BifidaMuscular Dystrophies+1 more
McGill University 8
Testing effectiveness (Phase 2)Study completedNCT00296621
What this trial is testing

Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Assistance Publique - Hôpitaux de Paris 30
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