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Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Recruitingn/aAges 5 Years - 62 YearsMaleUniversity of Florida
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In plain words

What the trial is, and whether it could suit you

The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD). The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with DMD with muscles of healthy individuals of the same age and monitor disease progression in those with DMD over a 5-10 year period. The amount of muscle damage and fat that the investigators measure will also be related to performance in daily activities, such as walking and the loss of muscle strength. In a small group of subjects the investigators will also assess the effect of corticosteroid drugs on the muscle measurements. Additionally, the investigators will map the progression of Becker MD following adults with this rare disease. The primar

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๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria for boys with DMD: 1\. Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with DMD based on: * clinical features with onset of symptoms before age five * elevated serum creatine kinase level or * absence of dystrophin expression, as determined by immunostain or western blot (\<2%) and/or DNA confirmation of a dystrophin mutation \*Subjects will not be excluded based on corticosteroid treatment or other clinical trials Inclusion Criteria for adults with Becker MD: 1. Ambulatory males (ages 18-62) without disease or injury to the lower extremities 2. Specific recruitment of a subset of individuals with deletion mutations in the dystrophin gene involving either exon 51 or exon 45. Inclusion Criteria for age matched controls for Becker MD subjects: 1\. Ambulatory males (ages 18-62) without disease or injury to the lower and/or upper extremities will be eligible to participate in this study Exclusion Criteria: 1. Males with a contraindication to an MR examination 2. Males with unstable medical problems 3. Males who are not able to cooperate during testing 4. Males with a secondary condition that may impact muscle metabolism, muscle function or functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease) 5. Daytime ventilation 6. Implantable Cardioverter Defibrillator- (ICD) or pace maker 7. Healthy boys/men who participate in competitive sports specific training in excess of 8 hours per week
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What it tests

  • Not listed
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Where

3 sites
University of Florida
Gainesville, United States
Oregon Health and Science University
Portland, United States
Children's Hospital of Philadelphia
Philadelphia, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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