803. 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.
805. 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.
808. Permethrin-Treated Baby Wraps for the Prevention of Malaria.
作者: Ross M Boyce.;Bonnie E Shook-Sa.;Ronnie Ndizeye.;Emmanuel Baguma.;Dana Giandomenico.;Caitlin A Cassidy.;Solomon Eshun.;Mark J Siedner.;Sarah G Staedke.;Moses Ntaro.;Jonathan J Juliano.;Raquel Reyes.;Edgar M Mulogo.
来源: N Engl J Med. 2025年393卷14期1399-1408页
Malaria remains a major cause of childhood death in sub-Saharan Africa. We leveraged the traditional practice of mothers carrying children on their backs in cloth wraps to assess whether treating the wraps with an insect repellent might provide a layer of protection against malaria.
820. CD19 CAR T-Cell Therapy in Multidrug-Resistant Ulcerative Colitis.
作者: Fabian Müller.;Raja Atreya.;Simon Völkl.;Michael Aigner.;Sascha Kretschmann.;Soraya Kharboutli.;Moritz Leppkes.;Selina Sitte.;Deike Strobel.;Arndt Hartmann.;Saurabh Mehandru.;Jean-Frederic Colombel.;Georg Schett.;Andreas Mackensen.;Markus F Neurath.
来源: N Engl J Med. 2025年393卷12期1239-1241页 |