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Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: ASCEND Trial Final Results 成人鞘磷脂酶缺乏症患者经Olipudase Alfa酶替代疗法治疗5年后临床结局持续改善:ASCEND试验最终结果 Wasserstein, Melissa P.; Hollak, Carla E.; Barbato, Antonio; Gallagher, Renata C.; Giugliani, Roberto; Guelbert, Norberto Bernardo; Hennermann, Julia B.; Ikezoe, Takayuki; Lachmann, Robin; Lidove, Olivier; Mabe, Paulina; Mengel, Eugen; Scarpa, Maurizio; Senates, Ebubekir; Tchan, Michel; Thurberg, Beth L.; Villarrubia, Jesus; Armstrong, Nicole M.; Bhushan, Shashi; Kim, Yong; Kumar, Monica 分享 收藏
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Erratum to Fabry disease during the last 20 years: Analysis of a cohort of 107 patients, and focus on the F113L variant [Rev Med Interne 46 (2025) 571-574] 勘误:过去20年中的法布里病:对107例患者的队列分析,并聚焦于F113L变异 [Rev Med Interne 46 (2025) 571-574] Lidove, Olivier; Subran, Benjamin; Montardi, Camille; Besset, Quentin; Mellot, Clara; Baba, Julien Aki; Azar, Chadi; London, Jonathan; Charron, Philippe; Caillaud, Catherine; Lamari, Foudil; Azevedo, Olga; Mauhin, Wladimir 分享 收藏
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Fabry disease during the last 20 years: Analysis of a cohort of 107 patients, and focus on the F113L variant 法布里病在过去20年:对107例患者的队列分析,并重点关注F113L变异体 Lidove, Olivier; Subran, Benjamin; Montardi, Camille; Besset, Quentin; Mellot, Clara; Baba, Julien Aki; Azar, Chadi; London, Jonathan; Charron, Philippe; Caillaud, Catherine; Lamari, Foudil; Azevedo, Olga; Mauhin, Wladimir 分享 收藏
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Final results of the ASCEND trial: Continued or sustained improvements in hepatosplenomegaly, respiratory outcomes, and lipid profile after 4 years of olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency Wasserstein, Melissa; Gallagher, Renata C.; Barbato, Antonio; Guigliani, Roberto; Guelbert, Norberto; Hennermann, Julia B.; Ikezoe, Takayuki; Lachmann, Robin; Lidove, Olivier; Mabe, Paulina; Mengel, Eugen; Scarpa, Maurizio; Senates, Ebubekir; Tchan, Michel; Villarrubia, Jesus; Thurberg, Beth L.; Armstrong, Nicole M.; Srivastav, Shruti; Wisniewska, Urszula; Kumar, Monica 分享 收藏
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Lysosphingolipid Quantitation in Plasma and Dried-Blood Spots Using Targeted High-Resolution Mass Spectrometry 血浆和干血斑中溶酶体鞘脂的靶向高分辨率质谱定量 Ducatez, Franklin; Mauhin, Wladimir; Ottaviani, Jules; Plichet, Thomas; Pilon, Carine; Lidove, Olivier; Barbey, Frederic; Perrichot, Regine; Vergnaud, Sabrina; Berger, Marc G.; Berger, Juliette; Belmatoug, Nadia; Nadjar, Yann; Lamari, Foudil; Noel, Esther; Marret, Stephane; Bekri, Soumeya; Tebani, Abdellah 分享 收藏
Acid sphingomyelinase deficiency in France: a retrospective survival study 法国酸性鞘磷脂酶缺乏症: 一项回顾性生存研究 Mauhin, Wladimir; Guffon, Nathalie; Vanier, Marie T.; Froissart, Roseline; Cano, Aline; Douillard, Claire; Lavigne, Christian; Heron, Benedicte; Belmatoug, Nadia; Uzunhan, Yurdagul; Lacombe, Didier; Levade, Thierry; Duvivier, Aymeric; Pulikottil-Jacob, Ruth; Laredo, Fernando; Pichard, Samia; Lidove, Olivier 分享 收藏
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Takotsubo syndrome occurring in systemic diseases: A French multicenter retrospective case-control study and literature review Culerrier, Julien; Terrier, Benjamin; Groh, Matthieu; Lopez-Sublet, Marilucy; Marie, Benjamin De Sainte; Falgarone, Geraldine; Lidove, Olivier; Mercie, Patrick; Mouthon, Luc; Meune, Christophe; Dhote, Robin 分享 收藏