"N Engl J Med"[journal] 检索结果

221. Engasertib in Hereditary Hemorrhagic Telangiectasia. Reply.

作者: Hanny Al-Samkari.;Damien Picard.;Hans-Jurgen Mager.
来源: N Engl J Med. 2026年394卷8期825-826页

222. Engasertib in Hereditary Hemorrhagic Telangiectasia.

作者: Deku Liang.;Yonghong Lin.;Li He.
来源: N Engl J Med. 2026年394卷8期825页

223. Beta-Blockers after Myocardial Infarction. Reply.

作者: John Munkhaugen.;Dan Atar.;Eva Prescott.
来源: N Engl J Med. 2026年394卷8期824-825页

224. Beta-Blockers after Myocardial Infarction. Reply.

作者: Borja Ibanez.;Roberto Latini.;Xavier Rossello.
来源: N Engl J Med. 2026年394卷8期823-824页

225. Beta-Blockers after Myocardial Infarction.

作者: Joseph J Zieminski.;Malcolm R Bell.
来源: N Engl J Med. 2026年394卷8期822-823页

226. Beta-Blockers after Myocardial Infarction.

作者: Marco Gamardella.;Giuseppe Ciconte.;Carlo Pappone.
来源: N Engl J Med. 2026年394卷8期822页

227. Beta-Blockers after Myocardial Infarction.

作者: Dídac Llop.;Marta Turu.;Carles Llop.
来源: N Engl J Med. 2026年394卷8期821页

228. Beta-Blockers after Myocardial Infarction.

作者: Kuan-Yu Chi.;Sridhar Mangalesh.;Michael G Nanna.
来源: N Engl J Med. 2026年394卷8期820-821页

229. Beta-Blockers after Myocardial Infarction.

作者: Alaa Rahhal.;Mohammed Al Hijji.;Abdul Rahman Arabi.
来源: N Engl J Med. 2026年394卷8期820页

230. Revival of Ethionamide by Alpibectir.

作者: Michel Pieren.;Glenn E Dale.;Marc Gitzinger.;Simon Tiberi.;Sophie L Penman.;Modesto J Remuiñán.;Ilse R Dubbelboer.;Ulrika S H Simonsson.;Caryn M Upton.;Thabo Mabuka.;Andreas H Diacon.
来源: N Engl J Med. 2026年394卷8期818-819页

231. One Pivotal Trial, the New Default Option for FDA Approval - Ending the Two-Trial Dogma.

作者: Vinay Prasad.;Martin A Makary.
来源: N Engl J Med. 2026年394卷8期815-817页

232. A Trial of an Antibody-Oligonucleotide Conjugate.

作者: Laura P W Ranum.
来源: N Engl J Med. 2026年394卷8期811-814页

233. Gene Therapy for Nephropathic Cystinosis.

作者: Olivier Devuyst.
来源: N Engl J Med. 2026年394卷8期809-810页

234. Biting the Tail of CAR-T for Relapsed Multiple Myeloma.

作者: Rakesh Popat.
来源: N Engl J Med. 2026年394卷8期807-809页

235. Case 6-2026: A 91-Year-Old Man with Shortness of Breath, Weight Loss, and Eosinophilia.

作者: Pavan K Bendapudi.;Maximiliano Klug.;Sharl S Azar.;Mandakolathur R Murali.;Ayman Al Jurdi.
来源: N Engl J Med. 2026年394卷8期794-805页

236. Peritoneal Coccidioidomycosis.

作者: Hayato Mitaka.;Michelle A Barron.
来源: N Engl J Med. 2026年394卷8期793页

237. Patient-Controlled Analgesia.

作者: James S Cho.;Ryan D'Souza.;David Hao.
来源: N Engl J Med. 2026年394卷8期e12页

238. An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1.

作者: Nicholas E Johnson.;Li-Jung Tai.;Johanna I Hamel.;John W Day.;Jeffrey M Statland.;Payam Soltanzadeh.;Sankarasubramoney H Subramony.;Charles A Thornton.;W David Arnold.;Matthew Wicklund.;Miriam L Freimer.;Kate Eichinger.;Jeanne Dekdebrun.;Chao-Yin Chen.;Varun Goel.;Bradley McEvoy.;Yiming Zhu.;Steven G Hughes.;Elizabeth J Ackermann.;Arthur A Levin.
来源: N Engl J Med. 2026年394卷8期763-772页

Myotonic dystrophy type 1 is a rare, dominantly inherited, progressive, disabling, neuromuscular disease that leads to decreased life expectancy and has no approved therapies. The disease is caused by a trinucleotide repeat expansion in DMPK, which encodes myotonic dystrophy type 1 protein kinase and imparts a toxic gain of function to the transcribed messenger RNA (mRNA), resulting in dysregulated alternative splicing (missplicing). Delpacibart etedesiran (del-desiran [AOC 1001]) is a monoclonal antibody-oligonucleotide conjugate. The antibody component targets transferrin receptor 1, and the oligonucleotide component targets DMPK mRNA.

239. Hematopoietic Stem-Cell Gene Therapy for Cystinosis.

作者: Bruce A Barshop.;Edward D Ball.;Nadine Benador.;Doris Trauner.;Susan Phillips.;Ranjan Dohil.;Natalie A Afshari.;Sohini Roy.;Beatriz Campo Fernandes.;Donald Kohn.;Katayoon Shayan.;John K Everett.;Frederic D Bushman.;Julian Midgley.;Hong Liang.;Anne Sawyers.;Jon A Gangoiti.;Maulik Panchal.;Imama Ahmed.;Stephanie Cherqui.
来源: N Engl J Med. 2026年394卷8期753-762页

Cystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in CTNS, the gene encoding cystinosin, a lysosomal transmembrane cystine transporter. In patients with cystinosis, cystine accumulates within lysosomes in all organs. The cystine-depleting agent cysteamine delays but does not prevent disease progression.

240. Tobacco Cessation among Nondaily and Low-Intensity Smokers - Challenges and Opportunities in Latin America.

作者: Laura Llambi.;Paola Morello.
来源: N Engl J Med. 2026年394卷8期729-732页

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