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Sarepta Therapeutics, Inc. Message Board

  • ocracokeclam ocracokeclam Feb 4, 2013 9:48 AM Flag

    Grant Awarded For MRI studies on Eteplirsen Patients

    HACKENSACK, N.J., Feb. 1, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD of the University of Florida in Gainesville to test the ability of magnetic resonance imaging (MRI) to detect changes in the muscles of those living with Duchenne muscular dystrophy (Duchenne) who participated in the Sarepta phase II trial of Eteplirsen.

    "This supplemental funding from PPMD allowed us to very quickly take advantage of a trial in progress to see if MRI can detect changes in the amount and quality of muscle in response to an experimental treatment," said Dr. Vandenborne.

    Eteplirsen is an antisense oligonucleotide-based drug that allows skipping of exon 51 in the dystrophin gene. For approximately 13% of those with Duchenne, skipping exon 51 may lead to the production of a shorter, but functional dystrophin protein. Sarepta Therapeutics recently completed a phase II study of the drug in 12 participants and found evidence of dystrophin production and some improvement in the distance walked in six minutes.

    Although the six minute walk test has been used as a primary outcome in almost all of the current clinical trials for Duchenne, study sponsors are also interested in identifying "biomarkers" or other types of measurements that might indirectly predict which study participants are responding to a therapeutic intervention. If such biomarkers can be identified and demonstrated to be linked to functional benefits, such as walking ability, they may one day be used in place of functional tests or to add supporting evidence to those tests.

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