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doi: 10.1186/s12967-019-02196-9 532 U.S

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Kinnari Patel, Rockets president and chief operating officer, noted that this treatment option has substantial morbidity and mortality and may not be available in time for these children. RP-L201 holds the FDAs Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, Fast Track and Orphan Drug designations

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