未登录AAV delivery of GBA1 suppresses α-synuclein accumulation in Parkinson's disease models and restores functions in Gaucher's disease modelsAAV递送GBA1在帕金森病模型中抑制α-突触核蛋白积累，并在戈谢病模型中恢复功能
Okai, Takuro; Sato, Sho; Deshpande, Mugdha; Matsumoto, Shin-ichi; Nakayama, Miyu; Yamamoto, Syunsuke; Strack-Logue, Bettina; Hioki, Takeshi; Tanaka, Maiko; Proetzel, Gabriele .png)
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收藏Identification of effect modifiers using a stratified Mendelian randomization algorithmic framework使用分层孟德尔随机化算法框架识别效应修饰因子Man, Alice; Knusel, Leona; Graf, Josef; Lali, Ricky; Le, Ann; Di Scipio, Matteo; Mohammadi-Shemirani, Pedrum; Chong, Michael; Pigeyre, Marie; Kutalik, Zoltan; Pare, Guillaume 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：第3B阶段延续研究的中期分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：第3B期延续研究的中期分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗在先天性血栓性血小板减少性紫癜患者中的应用：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：来自第3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗在先天性血栓性血小板减少性紫癜患者中的应用：第3B期延续研究的中期分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：来自第3B期延续研究的中期分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：第3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗在先天性血栓性血小板减少性紫癜患者中的应用：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗在先天性血栓性血小板减少性紫癜患者中的应用：来自第3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：第3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：第3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：来自第3B阶段延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防治疗先天性血栓性血小板减少性紫癜患者：第3B阶段延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 RECOMBINANT ADAMTS13 PROPHYLAXIS IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA: INTERIM ANALYSIS FROM THE PHASE 3B CONTINUATION STUDY重组ADAMTS13预防在先天性血栓性血小板减少性紫癜患者中的应用：来自3B期延续研究的期中分析Coppo, Paul; Patwari, Parth; Mellgard, Bjorn; Li, Hong; Scully, Marie; Matsumoto, Masanori; Windyga, Jerzy; Ortel, Thomas L.; Cataland, Spero; Knoebl, Paul; Yu, Ziqiang; Antun, Ana; Ibrahimi, Sami; Criger, Doug; Wang, Linda T. 分享 收藏 
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