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226. Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
作者: Francesca Fumagalli.;Valeria Calbi.;Vera Gallo.;Alberto Andrea Zambon.;Salvatore Recupero.;Francesca Ciotti.;Marina Sarzana.;Maddalena Fraschini.;Stefano Scarparo.;Fabiola De Mattia.;Simona Miglietta.;Clelia Pierini.;Matias Soncini.;Francesco Morena.;Eugenio Montini.;Federica Barzaghi.;Giulia Consiglieri.;Francesca Ferrua.;Maddalena Migliavacca.;Francesca Tucci.;Elena Sophia Fratini.;Alessia Ippolito.;Paolo Silvani.;Maria Rosa Calvi.;Alessandra Clerici.;Ambra Corti.;Marcella Facchini.;Sara Locatelli.;Mara Sangalli.;Stefano Zancan.;Federica Miotto.;Maria Grazia Natali Sora.;Cristina Baldoli.;Sabata Martino.;Angélica Córdoba-Claros.;Sean L Moro.;Nicholas D Gollop.;Jeff Abate.;Muska N Yarzi.;Philippa Nutkins.;Andrew Shenker.;Mattia Calissano.;Jean Brooks.;Alan Richardson.;Laura Campbell.;Massimo Filippi.;Luigi Naldini.;Maria Pia Cicalese.;Fabio Ciceri.;Maria Ester Bernardo.;Alessandro Aiuti.
来源: N Engl J Med. 2025年392卷16期1609-1620页
Metachromatic leukodystrophy (MLD) is an ultrarare, severe lysosomal storage disorder caused by a deficiency of arylsulfatase A (ARSA).
227. Tumor-Infiltrating Clonal Hematopoiesis.
作者: Oriol Pich.;Elsa Bernard.;Maria Zagorulya.;Andrew Rowan.;Constandina Pospori.;Ramy Slama.;Hector Huerga Encabo.;Jennifer O'Sullivan.;Despoina Papazoglou.;Panayiotis Anastasiou.;Chrysante S Iliakis.;Sally-Ann Clark.;Krijn K Dijkstra.;Vittorio Barbè.;Chris Bailey.;Aaron J Stonestrom.;Katey S S Enfield.;Mary Green.;Charlotte K Brierley.;Alastair Magness.;David R Pearce.;Robert E Hynds.;Rija Zaidi.;Jayant K Rane.;Ángel F Álvarez-Prado.;Kerstin Thol.;Rachel Scott.;Supreet Kaur Bola.;Elena Hoxha.;Steve K Harris.;Karl S Peggs.;Sergio A Quezada.;Allan Hackshaw.;Simone Zaccaria.;Johanna A Joyce.;Ilaria Malanchi.;Michael F Berger.;Mariam Jamal-Hanjani.;Andreas Wack.;Julian Downward.;William Grey.;Cristina Lo Celso.;Eva Grönroos.;Charles M Rudin.;Adam J Mead.;Dominique Bonnet.;Elli Papaemmanuil.;Charles Swanton.
来源: N Engl J Med. 2025年392卷16期1594-1608页
Clonal hematopoiesis of indeterminate potential (CHIP) is an age-related condition associated with increased mortality among patients with cancer. CHIP mutations with high variant-allele frequencies can be detected in tumors, a phenomenon we term tumor-infiltrating clonal hematopoiesis (TI-CH). The frequency of TI-CH and its effect on tumor evolution are unclear.
228. Efficacy of Baloxavir Treatment in Preventing Transmission of Influenza.
作者: Arnold S Monto.;Klaus Kuhlbusch.;Corrado Bernasconi.;Bin Cao.;Herman Avner Cohen.;Emily Graham.;Aeron C Hurt.;Laurie Katugampola.;Takashi Kamezawa.;Adam S Lauring.;Barry McLean.;Takahiro Takazono.;Andreas Widmer.;Steffen Wildum.;Benjamin J Cowling.
来源: N Engl J Med. 2025年392卷16期1582-1593页
Baloxavir marboxil (baloxavir) rapidly reduces influenza virus shedding, which suggests that it may reduce transmission. Studies of treatment with neuraminidase inhibitors have not shown sufficient evidence that they prevent transmission to contacts.
229. Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis.
作者: James D Chalmers.;Pierre-Régis Burgel.;Charles L Daley.;Anthony De Soyza.;Charles S Haworth.;David Mauger.;Michael R Loebinger.;Pamela J McShane.;Felix C Ringshausen.;Francesco Blasi.;Michal Shteinberg.;Kevin Mange.;Ariel Teper.;Carlos Fernandez.;Migdalia Zambrano.;Chunpeng Fan.;Xiangmin Zhang.;Mark L Metersky.; .
来源: N Engl J Med. 2025年392卷16期1569-1581页
In bronchiectasis, neutrophilic inflammation is associated with an increased risk of exacerbations and disease progression. Brensocatib, an oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP-1), targets neutrophil serine proteases, key mediators of neutrophilic inflammation.
230. Lorundrostat Efficacy and Safety in Patients with Uncontrolled Hypertension.
作者: Luke J Laffin.;Branko Kopjar.;Carrie Melgaard.;Kathy Wolski.;Jessica Ibbitson.;Shivani Bhikam.;Matthew R Weir.;Elizabeth O Ofili.;Reena Mehra.;James M Luther.;Debbie L Cohen.;Ashish Sarraju.;Michael J Wilkinson.;John M Flack.;David Rodman.;Steven E Nissen.; .
来源: N Engl J Med. 2025年392卷18期1813-1823页
Aldosterone dysregulation contributes to hypertension. Lorundrostat is an aldosterone synthase inhibitor, but data on its efficacy and safety in patients with hypertension are limited.
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