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The study was only in 12 patients, the drug will only help 13% of muscular dystrophy patients, the company may have to do more work before an FDA approval comes.
FORBES: The Breathtaking Slide From Sarepta's Muscular Dystrophy Study
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He persuaded a patient group, Parent Project Muscular Dystrophy, to grant a small amount of cash for preclinical work on the PTC medicine.
FORBES: Stopping the Nonsense
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Moreover, while the patient base for Duchenne may be too small to spark a billion-dollar drug, the PTC chemical could work against similar underlying gene flaws in other disorders such as cystic fibrosis, hemophilia and spinal muscular atrophy.
FORBES: Stopping the Nonsense
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Ms. von Rydingsvard's work, he said, has a strength and heroicism that should complement the Barclays Center's muscular architecture, and beckon people emerging from the subway at the corner of Atlantic and Flatbush avenues.
WSJ: 'Humanity' in Barclays Center's New Sculpture
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Hemsworth, whose only other notable work was a Kirk's Dad in last summer's Star Trek reboot, looks like a muscular guy.
FORBES: Magazine Article