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172. Sotatercept for Pulmonary Arterial Hypertension within the First Year after Diagnosis.
作者: Vallerie V McLaughlin.;Marius M Hoeper.;David B Badesch.;H Ardeschir Ghofrani.;J Simon R Gibbs.;Mardi Gomberg-Maitland.;Ioana R Preston.;Rogerio Souza.;Aaron B Waxman.;Grzegorz Kopeć.;Gisela Meyer.;Karen M Olsson.;Wei Fu.;Yaru Shi.;Barry Miller.;Samuel S Kim.;Harald S Mackenzie.;Michela Brambatti.;Mahesh J Patel.;Joerg Koglin.;Alexandra G Cornell.;Marc Humbert.; .
来源: N Engl J Med. 2025年393卷16期1599-1611页
Sotatercept, an activin-signaling inhibitor, reduces morbidity and mortality among patients with long-standing pulmonary arterial hypertension. Its effects in patients with pulmonary arterial hypertension within the first year after diagnosis are unclear.
177. Hypertonic Saline or Carbocisteine in Bronchiectasis.
作者: Judy M Bradley.;Brenda O'Neill.;Daniel F McAuley.;James D Chalmers.;Anthony De Soyza.;Adam T Hill.;Mary Carroll.;Michael R Loebinger.;Jamie Duckers.;Mike Clarke.;Rebecca H McLeese.;Kathryn Ferguson.;Andrew Jackson.;Christina Campbell.;Clíona McDowell.;Ashley Agus.;John Norrie.;Fiona Copeland.;Damian G Downey.;Rory Convery.;Martin Kelly.;William Flight.;Nick P Talbot.;John R Hurst.;John Steer.;Muhammad Anwar.;Mitra Shahidi.;Timothy Gatheral.;Mohamed Etumi.;Anita L Sullivan.;Andreea Alina Ionescu.;Veeresh Patil.;Milan Bhattacharya.;Steven Caskey.;Denise Cosgrove.;Conor Hagan.;Amelia Shoemark.;Terence McManus.;Gareth Davies.;J Stuart Elborn.; .
来源: N Engl J Med. 2025年393卷16期1565-1577页
Bronchiectasis guidelines are inconsistent with regard to the effectiveness of mucoactive agents, and their use varies geographically. Large trials are needed to assess safety and effectiveness.
179. Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy.
作者: Julian D Gillmore.;Ed Gane.;Jörg Täubel.;Björn Pilebro.;Andoni Echaniz-Laguna.;Justin Kao.;William Litchy.;Safi Shahda.;Alexandra Haagensen.;Liron Walsh.;Derek Smith.;Jessica Kachadourian.;Jonathan H Ward.;David Lebwohl.;Peijuan Zhu.;Yuanxin Xu.;Adia Leung.;Alison Sonderfan.;David E Gutstein.;Garen Manvelian.;David Adams.
来源: N Engl J Med. 2025年393卷14期1375-1386页
Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, multisystem, progressive, debilitating, and fatal disease characterized by tissue deposition of misfolded transthyretin (TTR) in peripheral nerves. Nexiguran ziclumeran (nex-z) is an investigational in vivo therapy based on CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease) that is designed to reduce serum TTR levels through selective inactivation of TTR in the liver.
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