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Chenodiol, Pengobatan Pertama untuk Cerebrotendinous Xanthomatosis, Mendapat Persetujuan FDA
Majalah Farmasetika – FDA telah menyetujui tablet chenodiol (Ctexli; Mirum Pharmaceuticals) untuk pengobatan cerebrotendinous xanthomatosis (CTX). Dengan persetujuan ini, chenodiol menjadi obat pertama yang disetujui FDA untuk mengobati penyakit penyimpanan lipid yang sangat langka ini. Apa Itu CTX? CTX adalah gangguan metabolik genetik langka yang disebabkan oleh mutasi pada gen CYP27A1,…
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