罕见病-话题@MedSci

#罕见病#

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梅斯话题小助手

罕见病

2020-05-29

lifefamily (审核中...)

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#罕见病#泡泡男孩，因为电影而被人知晓

EMA授予泡泡男孩病两项基因治疗药物的孤儿药资格

2020-12-02

lifefamily (审核中...)

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#黏多糖贮积症#好象不少见哦，是一种较多的#罕见病#

黏多糖贮积症（Mucopolysaccharidsis，MPS）基因治疗计划取得积极中期数据

2020-12-02

lifefamily (审核中...)

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#重症肌无力#的#罕见病#

JAMA Neurol：利妥昔单抗治疗新发全身性重症肌无力

2020-12-02

lifefamily (审核中...)

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#罕见病#这是目前看到最经典，最全面的信息，收藏！！

121种“常见的”罕见病名录，以及我国主要罕见病介绍，请速收藏并转发

2020-12-02

lifefamily (审核中...)

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#戈谢病#是#罕见病#，目前针对罕见病，路子越来越好了

罕见病日｜从戈谢病治疗20年看罕见病救助，困难究竟在哪？

2020-12-02

heli0118 (审核中...)

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#罕见病##肾上腺脑白质营养不良#

Bluebird突破性基因疗法Lenti-D申请上市！改善致死罕见病CALD患者长期生存结局

2020-12-02

heli0118 (审核中...)

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#红斑性肢痛症#是一种#罕见病#

关注罕见病“红斑性肢痛症”：FDA授予ATX01孤儿药称号

2020-12-02

lovetcm (审核中...)

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#RNAi疗法#多用于#罕见病#，如这个急性#肝卟啉症#

FDA批准Alnylam的RNAi疗法givosiran治疗罕见遗传病——急性肝卟啉症

2020-11-20

1381abdab4m (审核中...)

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#罕见病##亨特综合征#，即#II型黏多糖贮积症#药物艾度硫酸酯酶β注射液上市，祝贺！

亨特综合征新药「艾度硫酸酯酶β注射液」在中国获批

2020-09-05

docwu2019 (审核中...)

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#罕见病##罕见病新药#

Arimoclomol治疗丙型尼曼-皮克病：已完成新药申请

2020-08-13

lovetcm (审核中...)

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这是非常少见的肺内肿瘤。#罕见病#

原发性肺滑膜肉瘤1例

2020-06-23

lovetcm (审核中...)

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#罕见病#mark，Still病

FDA批准Still病的首款药物：诺华的白细胞介素-1单抗canakinumab

2020-06-17

lovetcm (审核中...)

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#罕见病#很多罕见病在国内都是大量患者群体

盘点——那些越来越不罕见的“罕见病”

2020-06-07

lovetcm (审核中...)

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#罕见病#有潜力

FDA授予AAV基因疗法PBGM01"孤儿药"称号，治疗婴儿GM1神经节苷脂贮积症

2020-06-07

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