未登录 Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3trial 米塔匹泛在依赖输血的α-地中海贫血或β-地中海贫血成人中的有效性和安全性（ENERGIZE-T）：一项双盲、随机、多中心、安慰剂对照、III期临床试验 Cappellini, Maria Domenica; Sheth, Sujit; Taher, Ali; Al-Samkari, Hanny; Antmen, Ali Bulent; Beneitez, David; Cannas, Giovanna; Coates, Thomas; Czapla, Lauren; Dahlin, Jayme L.; Estepp, Jeremie H.; Feenstra, Elizabeth; Georgiev, Pencho; Gheuens, Sarah; Price, Gavrielle M.; Glenthoj, Andreas; Musallam, Khaled M.; Osman, Kareem; Porter, John B.; Shao, Hui; Uhlig, Katrin; Beers, Eduard J. van; Viprakasit, Vip; Kuo, Kevin H. M.; Kattamis, Antonis 分享 收藏
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Pharmacogenomic and Clinical Predictors of Deferasirox Response in Transfusion-Dependent Thalassemia Identified Using Whole-Genome Sequencing Yampayon, Kittika; Sakares, Watchara; Pitinanon, Krittin; Limwongse, Chanin; Srichairatanakool, Somdet; Koonyosying, Pimpisid; Sukasem, Chonlaphat; Triparn, Kwankom; Piriyapongsa, Jittima; Ekwattanakit, Supachai; Viprakasit, Vip; Yodsurang, Varalee 分享 收藏
Bridging Pharmacokinetics and Pharmacodynamics: A PBPK/PD Model-Based Approach for Deferasirox Dosing in Transfusion-Dependent Thalassemia 连接药代动力学与药效学：一种基于PBPK/PD模型的地拉罗司在输血依赖型地中海贫血中的给药方案 Sakares, Watchara; Udomnilobol, Udomsak; Pan, Xian; Yampayon, Kittika; Ekwattanakit, Supachai; Viprakasit, Vip; Limwongse, Chanin; Srichairatanakool, Somdet; Teerawonganan, Polsak; Kunsa-ngiem, Sumate; Khaowroongrueng, Vipada; Techatanawat, Isariya; Prueksaritanont, Thomayant; Yodsurang, Varalee 分享 收藏
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Bioanalytical method for NAD+ detection in blood plasma utilizing solution-phase Candida boidinii formate dehydrogenase and electrochemical detection Taron, Wichit; Kasemphong, Tharinda; Sunon, Pachanuporn; Kaewket, Keerakit; Kamonsutthipaijit, Nuntaporn; Ketudat-Cairns, James R.; Bhakdisongkhram, Gun; Tulalamba, Warut; Sanguansuk, Supakmongkon; Viprakasit, Vip; Ngamchuea, Kamonwad 分享 收藏
Pharmacogenomic landscape of the Thai population from genome sequencing of 949 individuals Piriyapongsa, Jittima; Chumnumwat, Supatat; Kaewprommal, Pavita; Triparn, Kwankom; Suvichapanich, Supharat; Udomsinprasert, Wanvisa; Jittikoon, Jiraphun; Shaw, Philip J.; Nakhonsri, Vorthunju; Ngamphiw, Chumpol; Wangkumhang, Pongsakorn; Pithukpakorn, Manop; Roothumnong, Ekkapong; Wiboonthanasarn, Supakit; Kuptanon, Chulaluck; Jinawath, Natini; Porntaveetus, Thantrira; Suriyaphol, Prapat; Viprakasit, Vip; Pisitkun, Prapaporn; Kantaputra, Piranit; Tim-Aroon, Thipwimol; Wattanasirichaigoon, Duangrurdee; Sura, Thanyachai; Suphapeetiporn, Kanya; Sripichai, Orapan; Khongphatthanayothin, Apichai; Fucharoen, Suthat; Ngamphaiboon, Nuttapong; Shotelersuk, Vorasuk; Mahasirimongkol, Surakameth; Tongsima, Sissades 分享 收藏
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Compliance and clinical benefit of deferasirox granule and dispersible tablet formulation in pediatric patients with transfusional iron overload: in a randomized, open-label, multicenter, phase II study Taher, Ali T.; Wali, Yasser; Cruz, Maria Cecilia; Charoenkwan, Pimlak; Aydinok, Yesim; Werner, Olena; Govindaraju, Sameera; Romen, Fabian; Viprakasit, Vip 分享 收藏
Bone mineral density in adult patients with pyruvate kinase deficiency on long-term mitapivat treatment 长期接受mitapivat治疗的丙酮酸激酶缺乏症成年患者的骨矿物质密度 Al-Samkari, Hanny; Grace, Rachael F.; Glenthoj, Andreas; Andres, Oliver; Barcellini, Wilma; Galacteros, Frederic; Kuo, Kevin H. M.; Layton, D. Mark; Morado, Marta; Viprakasit, Vip; Tai, Feng; Urbstonaitis, Rolandas; Morales, Jaime; Mcgee, Bryan; Beers, Eduard J. van 分享 收藏
Patient preference for deferasirox film-coated versus dispersible tablet formulation: a sequential-design phase 2 study in patients with thalassemia Viprakasit, Vip; Hamdy, Mona M. M.; Hassab, Hoda M. A.; Sherief, Laila M. M.; Al-Bagshi, Muneer; Khattab, Mohammed; Chuncharunee, Suporn; Dung, Phu Chi; Kupesiz, Alphan; Shekhawat, Ankita; Sonawane, Yamini; Perez, Laura Torres; Slader, Cassandra; Taher, Ali T. T. 分享 收藏
The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry Schwab, Marisa E.; Lianoglou, Billie R.; Gano, Dawn; Velez, Juan Gonzalez; Allen, Isabel E.; Arvon, Regina; Baschat, Ahmet; Bianchi, Diana W.; Bitanga, Melissa; Bourguignon, Anne; Brown, Richard N.; Chen, Bruce; Chien, May; Davis-Nelson, Shareece; de Laat, Monique W. M.; Ekwattanakit, Supachai; Gollin, Yvonne; Hirata, Greigh; Jelin, Angie; Jolley, Jennifer; Meyer, Paul; Miller, Jena; Norton, Mary E.; Ogasawara, Keith K.; Panchalee, Tachjaree; Schindewolf, Erica; Shaw, Steven W.; Stumbaugh, Tammy; Thompson, Alexis A.; Towner, Dena; Tsai, Pai-Jong Stacy; Viprakasit, Vip; Volanakis, Emmanuel; Zhang, Li; Vichinsky, Elliott; MacKenzie, Tippi C. 分享 收藏
Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency Al-Samkari, Hanny; Grace, Rachael F.; Glenthoj, Andreas; Andres, Oliver; Barcellini, Wilma; Galacteros, Frederic; Kuo, Kevin H. M.; Layton, D. Mark; Morado Arias, Marta; Viprakasit, Vip; Dong, Yan; Tai, Feng; Hawkins, Peter; Gheuens, Sarah; Morales-Arias, Jaime; Gilroy, Keely S.; Porter, John B.; van Beers, Eduard J. 分享 收藏
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Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial Taher, Ali T.; Cappellini, Maria Domenica; Kattamis, Antonis; Voskaridou, Ersi; Perrotta, Silverio; Piga, Antonio G.; Filosa, Aldo; Porter, John B.; Coates, Thomas D.; Forni, Gian Luca; Thompson, Alexis A.; Tartaglione, Immacolata; Musallam, Khaled M.; Backstrom, Jay T.; Esposito, Oriana; Giuseppi, Ana Carolina; Kuo, Wen-Ling; Miteva, Dimana; Lord-Bessen, Jennifer; Yucel, Aylin; Zinger, Tatiana; Shetty, Jeevan K.; Viprakasit, Vip 分享 收藏
Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial Glenthoj, Andreas; van Beers, Eduard J.; Al-Samkari, Hanny; Viprakasit, Vip; Kuo, Kevin H. M.; Galacteros, Frederic; Chonat, Satheesh; Porter, John; Zagadailov, Erin; Xu, Rengyi; Oluyadi, Abdulafeez; Hawkins, Peter; Gheuens, Sarah; Beynon, Vanessa; Barcellini, Wilma 分享 收藏
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