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Results for “Neuromuscular; Disorder, Hereditary”

1,924 trials

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Showing 20 of 1,924 results

Not applicableUnknownNCT02614820
What this trial is testing

The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
General Hospital of Chinese Armed Police Forces 100
Large-scale testing (Phase 3)Study completedNCT05156320
What this trial is testing

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more
Scholar Rock, Inc. 188
Not applicableStudy completedNCT01689480
What this trial is testing

Prospective Study for 24-months of Physical Training Introduced in Lifestyle of Patients With FSHD : Tolerance, Sustainability and Efficiency of Unsupervised Training Program.

Who this might be right for
Muscular Dystrophy, Facioscapulohumeral
Centre Hospitalier Universitaire de Saint Etienne 15
Not applicableLooking for participantsNCT07423026
What this trial is testing

A Remote Study Using Technology to Assess Outcomes in DMD

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
University of Oxford 60
Not applicableLooking for participantsNCT07385443
What this trial is testing

The Spanish National Registry for Myotonic Dystrophy Type 1

Who this might be right for
Myotonic Dystrophy 1DM1Myotonic Dystrophy Type 1+2 more
Fundació Institut Germans Trias i Pujol 3,000
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
Early research (Phase 1)Ended earlyNCT02292225
What this trial is testing

Duvelisib With Obinutuzumab in Patients With CLL/SLL Previously Treated With a BTKi (SYNCHRONY)

Who this might be right for
Lymphocytic Leukemia, ChronicLymphoma, Small Lymphocytic
SecuraBio 3
Not applicableStudy completedNCT01313325
What this trial is testing

Hippotherapy to Improve the Balance of Children With Movement Disorders

Who this might be right for
Neuromuscular DiseasesCerebral PalsyDown Syndrome+2 more
Central Michigan University 16
Not applicableLooking for participantsNCT03882827
What this trial is testing

Natural History of Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Genethon 220
Testing effectiveness (Phase 2)Study completedNCT04396496
What this trial is testing

Treatment of POEMS Syndrome With Daratumumab

Who this might be right for
POEMS Syndrome
University of Arkansas 2
Not applicableUnknownNCT05249361
What this trial is testing

Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Samsung Medical Center 48
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Early research (Phase 1)Study completedNCT00451074
What this trial is testing

Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons

Who this might be right for
Duchenne Muscular Dystrophy
Nationwide Children's Hospital 12
Not applicableUnknownNCT05701397
What this trial is testing

Prevalence of Cardiac Amyloidosis in Patients Undergoing Surgery for Lumbar Spinal Stenosis.

Who this might be right for
Cardiac AmyloidosisSpinal Stenosis
Steen Hvitfeldt Poulsen 200
Not applicableStudy completedNCT06582537
What this trial is testing

LAMA2 Genetic Correction

Who this might be right for
MDC1A
Maastricht University 7
Testing effectiveness (Phase 2)Ended earlyNCT04264442
What this trial is testing

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

Who this might be right for
Facioscapulohumeral Muscular Dystrophy (FSHD)
Fulcrum Therapeutics 76
Not applicableUnknownNCT01777451
What this trial is testing

Whole Body Magnetic Resonance Imaging With Diffusion Weighted Imaging : Potential Role in Neurofibromatosis

Who this might be right for
Whole Body ImagingMagnetic Resonance ImagingNeurofibromatosis 1+2 more
Universitaire Ziekenhuizen KU Leuven 30
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableNo Longer AvailableNCT03317366
What this trial is testing

Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies

Who this might be right for
Proteus SyndromePIK3CA-Related Overgrowth Spectrum (PROS)Growth Disorders
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Early research (Phase 1)Ended earlyNCT04800822
What this trial is testing

PF-07284892 in Participants With Advanced Solid Tumors

Who this might be right for
Solid Tumor
Pfizer 53
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