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The U.S. Food and Drug Administration cleared a shortened version of a gene for a muscle protein to be used in 4- and 5-year-olds with muscular dystrophy.
The FDA OK'd the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition.
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First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids ... Updated: 3:28 AM CDT Jun 23, 2023 Editorial Standards ⓘ By MATTHEW PERRONE, AP Health Writer ...
First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids Share Updated: 3:28 AM CDT Jun 23, 2023 Infinite Scroll Enabled ...
The FDA OK'd the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition.