美國食品藥物管理局核准一項針對罕見免疫疾病的突破性基因療法
FDA approves breakthrough gene therapy for a rare immune disorder
2026年3月，美國食品藥物管理局（FDA）授予Kresladi加速批准，達成了一項歷史性里程碑。
In March 2026, the U.S.
這是一種開創性的基因療法，專為患有嚴重第一型白血球黏附缺乏症（LAD-I）的兒童所設計。
Food and Drug Administration (FDA) reached a historic milestone by granting accelerated approval to Kresladi, a groundbreaking gene therapy designed for children with severe leukocyte adhesion deficiency type I (LAD-I).
這種極為罕見的遺傳性疾病，係因ITGB2基因突變所致，會導致白血球無法對抗感染，且常在幼兒時期便致命。
This ultra-rare genetic disorder, caused by mutations in the ITGB2 gene, prevents white blood cells from fighting infections, often proving fatal in early childhood.
Kresladi是針對此病症首個獲批的療法，堪稱重大醫學突破。
Kresladi represents a major medical breakthrough as the first-ever approved treatment for this condition.
該療法由Rocket Pharmaceuticals公司開發，屬於自體（ㄗˋㄊㄧˇ）療法，意即利用病患自身的修飾幹細胞來恢復必要的免疫功能。
Developed by Rocket Pharmaceuticals, the therapy is autologous, meaning it uses a patient’s own modified stem cells to restore essential immune function.
透過重新導入健康的ITGB2基因副本，該療法能協助身體有效清除危險的細菌與真菌。
By reintroducing healthy copies of the ITGB2 gene, the treatment helps the body effectively clear dangerous bacteria and fungi.
由加州大學洛杉磯分校（UCLA）的唐納·科恩（Donald Kohn）博士所領導的臨床試驗顯示，病患的嚴重感染次數減少，且生物標記（ㄅㄧㄠ ㄐㄧˋ）也隨時間持續改善。
Clinical trials, led by Dr.
為表彰該公司致力於解決嚴峻兒科需求的努力，FDA亦授予該公司一張罕見兒科疾病優先審查憑證。
This one-time, potentially curative therapy now offers hope to families where no donor exists.
