欧盟委员会(EC)已批准Strimvelis用于腺苷脱氨酶(ADA)缺乏性重度联合免疫缺陷症(ADA-SCID)儿科患者。ADA-SCID是一种非常罕见的疾病,患者出生时便不具备健康、健全的免疫系统功能,导致对日常感染失去抵抗力。
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The Glaxo gene therapy program will focus on a severe immune deficiency called ADA-SCID, in which kids cannot make a crucial enzyme called adenosine deaminase that is needed to create immune system cells.
FORBES: Glaxo Brings Gene Therapy Back From The Dead
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