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Sarepta Therapeutics, Inc. Message Board

  • clarkst70 clarkst70 Feb 7, 2013 12:03 AM Flag

    cbs story boston

    BEVERLY (CBS) – A Massachusetts family is in a fight to save their son.

    They’re going to Washington to plead with the FDA to approve a drug that would give them some hope.

    When Max Gaudenzi was six years old, he played Little League, got around on his own and was like other kids.

    Max says, “I remember I use to swim and now I can’t anymore.”

    At 17, Max can’t even scratch his nose.

    “Well I can’t really take care of myself,” he says.

    “His friends are driving now and they have girlfriends now and they have a good social life,” says his dad Paul Gaudenzi. “It’s tough on Max.”

    It’s tough on his mom and dad too. Max has Duchenne muscular dystrophy.

    “There is no cure for it,” says his mom Natalie, “it’s a fatal disease.”

    There is no cure, but for the last two years, a drug in the testing phase has shown signs of progress.

    “If we could see some kind of gain from this drug, that simply he could reach his face or scratch an itch or feed himself, that would be a miracle to us,” says Natalie.

    The Gaudenzis are traveling to D.C. this weekend to try and convince lawmakers and the FDA to release the drug now.

    “The main thing is we want to see something happen, it’s difficult, but at least we have some hope,” said Paul.

    The drug being tested is Eteplirsen. The FDA will keep testing it for another three to six years, but families like the Gaudenzis say time is something they don’t have much of.

    “My fear is that our boys at this age will be too disabled or even alive to benefit from this drug,” said Natalie.

    This form of muscular dystrophy affects mostly boys. Many of them will join the Gaudenzis in Washington to persuade the FDA to put the drug’s approval on the fast track.

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