X连锁-话题@MedSci

#X连锁#

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X连锁

2020-05-29

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#X连锁#

X连锁无丙种球蛋白血症：症状与体征、病因、流行病学、诊断与治疗

2023-02-18

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#X连锁#

X连锁淋巴增生症：症状与体征，病因，流行病学，诊断与治疗

2022-09-29

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#X连锁#

ARD：SAT1中的功能丧失变异导致X连锁儿童期系统性红斑狼疮

2022-12-11

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X连锁肾上腺脑白质营养不良：症状与体征、病因、流行病学、诊断和治疗

2022-09-03

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#X连锁#

X连锁低磷血症（XLH）：新数据表明Crysvita对XLH患者具有长期益处

2021-12-09

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#X连锁#

Nat Commun：X连锁肌张力障碍-帕金森病中年龄相关遗传修饰因子的鉴定

2021-07-05

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关注罕见病“红细胞生成性原卟啉症及X连锁卟啉症”：已启动MT-7117全球III期临床试验

2020-11-20

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NMPA批准突破性罕见病药布罗索尤单抗上市，治疗X连锁低磷血症（遗传佝偻病）

2021-03-24

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#X连锁#

Nat Med：慢病毒基因疗法治疗X连锁慢性肉芽肿病

2021-07-26

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欧洲药品管理局：授予MB-107慢病毒基因疗法治疗X连锁严重综合免疫缺陷病的先进疗法分类

2021-01-14

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#X连锁#

J Bone Miner Res：布洛舒单抗可改善X连锁低磷酸盐血症成人的骨软化情况

2021-05-19

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欧洲委员会（EC）批准CRYSVITA（burosumab）治疗老年人和成年人X连锁性低磷血症（XLH）

2021-02-23

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JCEM：X连锁性低磷血症的患病率和死亡率

2020-07-11

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#X连锁#

第24届世界肌肉协会年会：AT132治疗X连锁型肌管肌病的ASPIRO试验取得新阳性数据

2020-05-20

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#X连锁#

Neurology病例：见于成人起病的X连锁肾上腺脑白质营养不良的额叶受累

2020-08-15

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#X连锁#

FDA授予OTL-102孤儿药称号，用于治疗X连锁慢性肉芽肿（X-CGD）

2020-10-31

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Neurology病例：X连锁肾上腺脑白质营养不良：脊髓小脑变异型

2020-02-19

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#X连锁#

Neurology：见于成人起病的X连锁肾上腺脑白质营养不良的额叶受累

2019-11-02

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#X连锁#

Hum Genome Var：日本X连锁先天性视网膜劈裂患者的RS1基因新突变研究

2019-03-02

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#X连锁#

Lentivector基因疗法治疗X连锁严重联合免疫缺陷

2020-04-07

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