23. Phase 3 Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis.
作者: Bruce C Trapnell.;Yoshikazu Inoue.;Francesco Bonella.;Tisha Wang.;Cormac McCarthy.;Toru Arai.;Keiichi Akasaka.;Francesca Mariani.;Nesrin Mogulkoc.;Jin Woo Song.;Tomohisa Baba.;Stephane Jouneau.;Tadahisa Numakura.;Nesrin Öcal.;Florin Mihaltan.;Ali Ataya.;Elisabeth Bendstrup.;Ilaria Campo.;Brenna Carey.;Ross Arena.;Brian Robinson.;Rosanna Fleming.;Yasmine Wasfi.;Raymond Pratt.; .
来源: N Engl J Med. 2025年393卷8期764-773页
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease characterized by progressive surfactant accumulation and hypoxemia caused by autoantibodies against granulocyte-macrophage colony-stimulating factor (GM-CSF), which alveolar macrophages require to clear surfactant. Molgramostim is a formulation of inhaled recombinant human GM-CSF, but its efficacy and safety in patients with aPAP have not been studied sufficiently.
39. Risdiplam in Presymptomatic Spinal Muscular Atrophy.
作者: Richard S Finkel.;Laurent Servais.;Dmitry Vlodavets.;Edmar Zanoteli.;Maria Mazurkiewicz-Bełdzińska.;Yuh-Jyh Jong.;Aledie Navas-Nazario.;Mohammad Al-Muhaizea.;Alexandra P Q C Araujo.;Leslie Nelson.;Yi Wang.;Birgit Jaber.;Ksenija Gorni.;Heidemarie Kletzl.;Laura Palfreeman.;Michael Rabbia.;Dave Summers.;Eleni Gaki.;Kathryn R Wagner.;Paulo Fontoura.;Michelle A Farrar.;Enrico Bertini.; .
来源: N Engl J Med. 2025年393卷7期671-682页
Risdiplam, an oral pre-messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic disease are unclear.
|