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FDA grants orphan drug designation to potential treatment of STXBP1 developmental and epileptic encephalopathyThe treatment was developed by Capsida Biotherapeutics and called CAP-002. DEE caused by STXBP1 mutations is estimated to affect up to one in 26,000 children globally. It is associated with ...
The couple also has Max, 13, and Lena, 9. Their youngest child, Lena, was diagnosed in 2015 with STXBP1. A nurse told Snedecor she just had “bad luck,” after Snedecor had asked if Jack’s ...
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