MDA Grant Will Help Families in DMD Steroid Study

A new MDA grant will defray travel costs for participants in a large-scale trial to find the optimal corticosteroid regimen for boys with Duchenne MD

Robert Griggs is a professor of neurology and a longtime MDA research grantee at the University of Rochester (N.Y.).
Article Highlights:
  • The U.S. National Institutes of Health (NIH) is sponsoring a multinational, multiyear study to determine which treatment regimen (dosing schedule) most effectively balances the benefits and side effects of the corticosteroid drugs prednisone and deflazacort in boys with Duchenne muscular dystrophy.
  • The trial is now open at approximately 40 locations in several countries, including the U.S.
  • MDA has awarded a grant to Robert Griggs at the University of Rochester (N.Y.) so that travel and lodging costs will not be a barrier to North American families who want to participate in this trial.
by Margaret Wahl on October 24, 2013 - 2:00pm

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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