BMD: Participants Sought for Registry Associated with Gene Transfer Trial

In connection with a gene transfer clinical trial, investigators at Nationwide Children's Hospital in Columbus, Ohio, are seeking people with Becker muscular dystrophy (BMD) for participation in a patient registry.

The investigators say the registry will provide information that will:

Follistatin is a protein that promotes muscle growth and strength. In 2009, researchers showed that genes for the follistatin protein increased strength when injected into the leg muscles of monkeys.

The patient registry project — Clinical Outcomes Studies in Becker Muscular Dystrophy (BMD) and Sporadic Inclusion-Body Myositis (sIBM)— involves tests of muscle strength and function. (At this time, the patient registry is seeking only people with BMD, not sporadic inclusion-body myositis.)

Prospective participants in the registry must:

  • have a diagnosis of BMD, with weakness of the quadriceps (thigh) muscles;
  • have some difficulty getting out of chairs, climbing stairs or getting up from the floor;
  • be able to walk independently without the aid of a walker or cane; and
  • be able to cooperate for muscle strength testing.

Prospective participants in the registry must not:

  • have a history of liver disease or HIV infection;
  • have a history of angina or myocardial infarction within the past six months; or
  • be taking prednisone or other immunosuppressive drugs.

To participate in the registry

Contact Lindsay Alfano in Columbus, Ohio, at (614) 722-6881 or lindsay.alfano@nationwidechildrens.org.

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