Media Coverage
"I'm not that person anymore" Live Oak teen's life transformed by rare disease treatment
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Riverbend News
CIRM gets first approval of a one-and-done gene therapy
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Capitol Weekly
FDA approval makes Rocket Pharma gene therapy the first for ultra-rare immune disorder
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Medcity News
They didn't understand why their kids kept getting sick. After a rare diagnosis, a new gene therapy offered hope.
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CBS News