1182. Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
作者: Ulrike M Reiss.;Andrew M Davidoff.;Edward G D Tuddenham.;Pratima Chowdary.;Jenny McIntosh.;Vincent Muczynski.;Malo Journou.;Giulia Simini.;Lydia Ireland.;Saira Mohamed.;Anne Riddell.;Arnulfo J Pie.;Andrew Hall.;Alberto Quaglia.;Sarah Mangles.;Johnny Mahlangu.;Kristina Haley.;Michael Recht.;Yu-Min Shen.;Kathleen G Halka.;Gail Fortner.;Christopher L Morton.;Zhengming Gu.;Randall T Hayden.;Ellis J Neufeld.;Victoria I Okhomina.;Guolian Kang.;Amit C Nathwani.
来源: N Engl J Med. 2025年392卷22期2226-2234页
Adeno-associated virus (AAV)-mediated gene therapy has emerged as a promising treatment for hemophilia B. Data on safety and durability from 13 years of follow-up in a cohort of patients who had been successfully treated with scAAV2/8-LP1-hFIXco gene therapy are now available.
1186. Finerenone with Empagliflozin in Chronic Kidney Disease and Type 2 Diabetes.
作者: Rajiv Agarwal.;Jennifer B Green.;Hiddo J L Heerspink.;Johannes F E Mann.;Janet B McGill.;Amy K Mottl.;Julio Rosenstock.;Peter Rossing.;Muthiah Vaduganathan.;Meike Brinker.;Robert Edfors.;Na Li.;Markus F Scheerer.;Charlie Scott.;Masaomi Nangaku.; .
来源: N Engl J Med. 2025年393卷6期533-543页
Limited evidence exists to support the simultaneous initiation of sodium-glucose cotransporter-2 inhibitors and finerenone, a nonsteroidal mineralocorticoid receptor antagonist, in persons with chronic kidney disease and type 2 diabetes.
1200. Bedaquiline Activity against Leprosy.
作者: Abdoulaye Fomba.;Fadima C Haidara.;Mamoudou Kodio.;Catherine Arama.;Emmanuelle Cambau.;Aurélie Chauffour.;Nicolas Veziris.;Bettina E Broeckling.;Alaine K Warren.;Bertrand Cauchoix.;Alexandre Alcaïs.;Laurent Marsollier.;Henri Assé.;Mary Jackson.;Samba Ousmane Sow.;Ousmane Faye.;Vincent Jarlier.;Stewart T Cole.;Charlotte Avanzi.;Alexandra Aubry.;Roch C Johnson.
来源: N Engl J Med. 2025年392卷21期2174-2176页 |