MDA Grant Will Help Families in DMD Steroid Study

Robert Griggs, a professor of neurology at the University of Rochester (N.Y.), has received an MDA grant of $237,316 over three years to support travel costs for North American participants in a large, multinational trial to determine which corticosteroid treatment regimen is best for children with Duchenne muscular dystrophy (DMD).

The corticosteroid drug prednisone has been found to prolong strength in boys with DMD, and another corticosteroid, deflazacort, may also be beneficial.

A few different corticosteroid treatment schedules have been used in DMD in an attempt to find the right balance between the benefits of corticosteroids and their notorious side effects, such as weight gain and bone loss. Which one is best, however, remains an open question.

About the corticosteroid trial

The new trial, which aims to add worldwide data to help settle the question of which corticosteroid regimen in DMD is optimal, is being sponsored by the U.S. National Institutes of Health (NIH) and is now open at most of its planned 39 locations in the United States, Canada, Germany, Italy and the United Kingdom.

Prospective participants must be 4 to 7 years old and meet other study criteria. (For details and updated information on which sites are recruiting new participants, see Finding the Optimum Regimen for Duchenne Muscular Dystrophy, or enter NCT01603407 into the search box at ClinicalTrials.gov.)

The investigators say the average family participating in this multiyear trial will need to make several overnight trips to a study site, possibly imposing a significant financial burden. The MDA grant for travel and lodging is meant to ease that burden.

More about corticosteroids in DMD

For more on corticosteroid treatment and research, read:

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more about clinical trials, see Being a Co-Adventurer. For a more refined list of clinical trials, visit
ClinicalTrials.gov, a registry of federally and privately supported clinical trials in the United States and around the world. Select the "Find Studies" tab, and follow the instructions to narrow down your search results.

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