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2928. Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera.
作者: Marina Kremyanskaya.;Andrew T Kuykendall.;Naveen Pemmaraju.;Ellen K Ritchie.;Jason Gotlib.;Aaron Gerds.;Jeanne Palmer.;Kristen Pettit.;Uttam K Nath.;Abdulraheem Yacoub.;Arturo Molina.;Samuel R Saks.;Nishit B Modi.;Frank H Valone.;Sarita Khanna.;Suneel Gupta.;Srdan Verstovsek.;Yelena Z Ginzburg.;Ronald Hoffman.; .
来源: N Engl J Med. 2024年390卷8期723-735页
Polycythemia vera is a chronic myeloproliferative neoplasm characterized by erythrocytosis. Rusfertide, an injectable peptide mimetic of the master iron regulatory hormone hepcidin, restricts the availability of iron for erythropoiesis. The safety and efficacy of rusfertide in patients with phlebotomy-dependent polycythemia vera are unknown.
2929. Biomarker Changes during 20 Years Preceding Alzheimer's Disease.
作者: Jianping Jia.;Yuye Ning.;Meilin Chen.;Shuheng Wang.;Hao Yang.;Fangyu Li.;Jiayi Ding.;Yan Li.;Bote Zhao.;Jihui Lyu.;Shanshan Yang.;Xin Yan.;Yue Wang.;Wei Qin.;Qi Wang.;Ying Li.;Jintao Zhang.;Furu Liang.;Zhengluan Liao.;Shan Wang.
来源: N Engl J Med. 2024年390卷8期712-722页
Biomarker changes that occur in the period between normal cognition and the diagnosis of sporadic Alzheimer's disease have not been extensively investigated in longitudinal studies.
2930. CD19 CAR T-Cell Therapy in Autoimmune Disease - A Case Series with Follow-up.
作者: Fabian Müller.;Jule Taubmann.;Laura Bucci.;Artur Wilhelm.;Christina Bergmann.;Simon Völkl.;Michael Aigner.;Tobias Rothe.;Ioanna Minopoulou.;Carlo Tur.;Johannes Knitza.;Soraya Kharboutli.;Sascha Kretschmann.;Ingrid Vasova.;Silvia Spoerl.;Hannah Reimann.;Luis Munoz.;Roman G Gerlach.;Simon Schäfer.;Ricardo Grieshaber-Bouyer.;Anne-Sophie Korganow.;Dominique Farge-Bancel.;Dimitrios Mougiakakos.;Aline Bozec.;Thomas Winkler.;Gerhard Krönke.;Andreas Mackensen.;Georg Schett.
来源: N Engl J Med. 2024年390卷8期687-700页
Treatment for autoimmune diseases such as systemic lupus erythematosus (SLE), idiopathic inflammatory myositis, and systemic sclerosis often involves long-term immune suppression. Resetting aberrant autoimmunity in these diseases through deep depletion of B cells is a potential strategy for achieving sustained drug-free remission.
2934. A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis.
作者: Gideon M Hirschfield.;Christopher L Bowlus.;Marlyn J Mayo.;Andreas E Kremer.;John M Vierling.;Kris V Kowdley.;Cynthia Levy.;Alejandra Villamil.;Alma L Ladrón de Guevara Cetina.;Ewa Janczewska.;Ehud Zigmond.;Sook-Hyang Jeong.;Yusuf Yilmaz.;Yiannis Kallis.;Christophe Corpechot.;Peter Buggisch.;Pietro Invernizzi.;Maria Carlota Londoño Hurtado.;Sandrin Bergheanu.;Ke Yang.;Yun-Jung Choi.;Daria B Crittenden.;Charles A McWherter.; .
来源: N Engl J Med. 2024年390卷9期783-794页
Effective treatments for patients with primary biliary cholangitis are limited. Seladelpar, a peroxisome proliferator-activated receptor delta agonist, has potential benefits.
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