Defining Endpoints in Becker Muscular Dystrophy

Open to males ages 8 years and up

For ages 8-16

• Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
• Genetic confirmation of an in-frame dystrophin mutation
• Ambulatory
• Willing and able to give informed consent and follow all procedures and requirements

For ages 17-older

• Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
• Genetic confirmation of a dystrophin mutation
• Willing and able to give informed consent and follow all procedures and requirements

For participants in the MRI substudy:

• Ambulatory, as defined as able to walk 10 meters without assistive devices (orthotics allowed)

For ages 8-16

• Out of frame dystrophin mutation
• Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
• Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
• >16 hours of ventilatory support
• Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.
• Under the age of 8 at time of enrollment
• For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)

For ages 17-older

• Loss of ambulation prior to age 16
• Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
• Less than 30% of the overall population will be non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
• >16 hours of ventilatory support
• For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)