Drugs

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The Food and Drug Administration has OK'd  the first U.S. treatment specifically for late-onset Pompe disease

posted on May 25, 2010 - 12:34pm
The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.
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"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval

posted on June 21, 2010 - 4:25pm
Update (Aug. 4, 2014): The U.S. Food and Drug Administration (FDA) has expanded the approval of Lumizyme so that it can now be used to treat Pompe disease patients of all ages, including children younger than age 8. See FDA Expands Approval of Drug to Treat Pompe Disease to Patients of All Ages; Removes Risk Mitigation Strategy Requirements, Aug. 1, 2014.
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The experimental drug SMT C1100 is designed to increase utrophin production as a treatment for Duchenne MD

posted on April 24, 2012 - 4:00am