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21. 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.
22. Dengue Suppression by Male Wolbachia-Infected Mosquitoes.
作者: Jue Tao Lim.;Chee-Seng Chong.;Chia-Chen Chang.;Diyar Mailepessov.;Borame Dickens.;Yee Ling Lai.;Lu Deng.;Caleb Lee.;Li Yun Tan.;Grace Chain.;Muhammad Faizal Zulkifli.;Jonathan Wee Kent Liew.;Kathryn Vasquez.;Man Ling Chau.;Youming Ng.;Vernon Lee.;Judith Chui Ching Wong.;Shuzhen Sim.;Cheong Huat Tan.;Lee Ching Ng.; .
来源: N Engl J Med. 2026年394卷12期1175-1183页
Wild-type female Aedes aegypti mosquitoes that mate with male A. aegypti mosquitoes that have been infected with the wAlbB strain of Wolbachia pipientis bacteria produce nonviable offspring owing to cytoplasmic incompatibility. Repeated releases of wolbachia-infected males can potentially suppress wild-type mosquito populations and reduce the risk of dengue virus infection.
23. AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.
作者: Connor J Lewis.;Precilla D'Souza.;Jean M Johnston.;Maria T Acosta.;Cristan Farmer.;Eva H Baker.;Anna Crowell.;Yoliann Mojica.;Sumaiya Ashraf.;Lisa Joseph.;Gilbert Vézina.;Zenaide Quezado.;Muhammad H Yousef.;Zeynep Vardar.;Mohammed Salman Shazeeb.;Manuela Corti.;Meghan Blackwood.;Kirsten Coleman.;Rita Batista.;Audrey Thurm.;Erika De Boever.;William A Gahl.;Barry J Byrne.;Terence R Flotte.;Xuntian Jiang.;Amanda L Gross.;Allison M Keeler.;Heather Gray-Edwards.;Douglas R Martin.;Miguel Sena-Esteves.;Cynthia J Tifft.
来源: N Engl J Med. 2026年394卷12期1184-1194页
GM1 gangliosidosis, caused by biallelic variants in GLB1, results from deficiency of lysosomal β-galactosidase, which degrades GM1 ganglioside. This fatal neurodegenerative disease currently has no effective therapy.
24. Palbociclib for Hormone-Receptor-Positive, HER2-Positive Advanced Breast Cancer.
作者: Otto Metzger.;Sumithra Mandrekar.;Shom Goel.;Joseph Gligorov.;Elgene Lim.;Eva Ciruelos.;Sibylle Loibl.;Travis Dockter.;Xavier Gonzàlez Farré.;Prudence A Francis.;Filipa Lynce.;Jane Lanzillotti.;Carter DuFrane.;Anna Wall.;Carrie Strand.;Ian Krop.;Ines Vaz-Luis.;Debu Tripathy.;Sherene Loi.;Aleix Prat.;Matthew Goetz.;Santiago Escrivá-de-Romaní.;David Porter.;Jennifer Spoenlein.;Daniel G Stover.;Sagar Sardesai.;Pierre Heudel.;Maria Koehler.;Cynthia Huang Bartlett.;Ariadna Holynskyj.;Prashanth Gopalakrishna.;Eric Gauthier.;Suzette Delaloge.;Kathy Miller.;Eric P Winer.;Luca Gianni.;Ann H Partridge.;Angela DeMichele.;Lisa A Carey.
来源: N Engl J Med. 2026年394卷5期451-462页
Dual anti-human epidermal growth factor receptor 2 (HER2) therapy plus chemotherapy followed by maintenance treatment with HER2-targeted and endocrine therapies is standard first-line treatment for hormone-receptor-positive, HER2-positive metastatic breast cancer. On the basis of preclinical and clinical data, the addition of palbociclib (a selective inhibitor of cyclin-dependent kinases 4 and 6) may overcome resistance to both endocrine and HER2-directed therapies.
25. A Randomized Trial of Tenecteplase in Acute Central Retinal Artery Occlusion.
作者: Stephen J Ryan.;Øystein Kalsnes Jørstad.;Mona Skjelland.;Maiju Pesonen.;Claus Z Simonsen.;Toke Bek.;Rolf Ankerlund Blauenfeldt.;Petra Ijäs.;Arja Laitinen.;Andrej Khanevski.;Jørgen Krohn.;Eyvind Rødahl.;Robin Lemmens.;Jelle Demeestere.;Catherine Cassiman.;Ingvild Nakstad.;Kristin Evensen.;Tiril Sandell.;Steffen Hamann.;Thomas C Truelsen.;Louisa M Christensen.;Sverre Rosenbaum.;Vaidas Matijošaitis.;Reda Žemaitienė.;Hanne Ellekjær.;Erlend Almaas.;Dordi Austeng.;Michael V Mazya.;Frank Traïsk.;Pauli Ylikotila.;Ulpu Salmi.;Kristian N Jenssen.;Håvard Lisether.;Cathrine Breivik.;Kristina Devik.;Lasse-Marius Elden Honningsvåg.;Jurgita Valaikienė.;Andrius Cimbalas.;Vetle Nilsen Malmberg.;Espen Anderson.;Ansar Roy.;Thor Håkon Skattør.;Kristian Lundsgaard Kraglund.;Christina Kefaloykos.;Inge Christoffer Olsen.;Peter Vanacker.;Daniel Strbian.;Morten C Moe.;Anne Hege Aamodt.; .
来源: N Engl J Med. 2026年394卷5期442-450页
Central retinal artery occlusion can result in permanent vision loss. Effective treatment is lacking.
26. Sacituzumab Govitecan plus Pembrolizumab for Advanced Triple-Negative Breast Cancer.
作者: Sara M Tolaney.;Evandro de Azambuja.;Kevin Kalinsky.;Sherene Loi.;Sung-Bae Kim.;Clinton Yam.;Bernardo Rapoport.;Seock-Ah Im.;Barbara Pistilli.;Wassim Mchayleh.;David W Cescon.;Junichiro Watanabe.;Manuel Alejandro Lara Bañuelas.;Ruffo Freitas-Junior.;Javier Salvador Bofill.;Maryam Afshari.;Dianna Gary.;Lu Wang.;Catherine Lai.;Peter Schmid.; .
来源: N Engl J Med. 2026年394卷4期354-366页
Triple-negative breast cancer is an aggressive breast cancer subtype, and there remains an unmet need to improve outcomes in patients with previously untreated, programmed death ligand 1 (PD-L1)-positive, locally advanced unresectable or metastatic triple-negative breast cancer.
27. CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia.
作者: Ruonan Li.;Hong Pan.;Lele Zhang.;Jiaxiu Ma.;Weiwang Li.;Zhen Gao.;Liwei Fang.;Linzhu Tian.;Yucan Shen.;Fei Yang.;Jingyu Zhao.;Neng Nie.;Jianping Li.;Wenyan Wang.;Xinan Pan.;Yu Lian.;Xingxin Li.;Guangxin Peng.;Liyun Li.;Xiao Yu.;Chun Xu.;Yanjie Liu.;Zhexiang Kuang.;Jinbo Huang.;Xin Zhao.;Meili Ge.;Lijun Liu.;Shuo Chen.;Yi Feng.;Alex H Chang.;Biping Deng.;Min Dai.;Lifang Huang.;Lulu Lv.;Yizhou Zheng.;Yuechen Luo.;Haiqing Xiong.;Jun Shi.
来源: N Engl J Med. 2026年394卷3期253-267页
In patients with autoimmune hemolytic anemia (AIHA), the risk of relapse is high owing to persistent autoreactive B-cell activity. Multirefractory AIHA is a more advanced stage of disease that is defined by a lack of response to at least three lines of therapy. CD19-directed chimeric antigen receptor (CAR) T-cell therapy results in profound B-cell depletion and may be a useful approach to achieving drug-free remission in multirefractory AIHA.
28. Fremanezumab in Children and Adolescents with Episodic Migraine.
作者: Andrew D Hershey.;Christina L Szperka.;Piero Barbanti.;Patricia Pozo-Rosich.;Petra Bittigau.;Steve Barash.;Juline Bryson.;Yoel Kessler.;Yael Carmeli Schwartz.;Verena Ramirez Campos.;Xiaoping Ning.
来源: N Engl J Med. 2026年394卷3期243-252页
Fremanezumab, a humanized monoclonal antibody that selectively targets calcitonin gene-related peptide, is approved for the prevention of migraine in adults. Evidence from randomized, controlled trials in children and adolescents is needed.
29. An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.
作者: Joseph Muenzer.;Barbara K Burton.;Paul Harmatz.;Deepa S Rajan.;Simon A Jones.;Johanna M P van den Hout.;John J Mitchell.;Akhil Bhalla.;Natalie J Engmann.;Imanol Zubizarreta.;Wei Dong.;Fabian Model.;Ryan J Watts.;Matthew D Troyer.;Peter S Chin.;Carole Ho.
来源: N Engl J Med. 2026年394卷1期39-50页
Tividenofusp alfa, comprising iduronate-2-sulfatase fused to an engineered transferrin receptor-binding Fc domain, has been developed to treat neurologic and peripheral manifestations of mucopolysaccharidosis type II (MPS II), a rare lysosomal disorder causing progressive multisystem and neurologic decline.
30. Trial of High-Dose Oral Rifampin in Adults with Tuberculous Meningitis.
作者: David B Meya.;Fiona V Cresswell.;Biyue Dai.;Nicole Engen.;Kogieleum Naidoo.;Ahmad Rizal Ganiem.;Darma Imran.;Mable Kabahubya.;Richard J Lessells.;Vycke Yunivita.;Riwanti Estiasari.;Lillian Tugume.;Bongeka Hlabisa.;Media Yuni Kurniawati.;Noveline Sagita.;Enock Kagimu.;Kartika Maharani.;Jane Gakuru.;Maula N Gaharu.;Timothy Mugabi.;Sarah Kimuda.;Suzan Namombwe.;Lindsey Te Brake.;Rob Aarnoutse.;Elin M Svensson.;Ananta S Bangdiwala.;Sylvia Namanda.;Nathan C Bahr.;Abdu K Musubire.;Mahomed Yunus Suleman Moosa.;Raph L Hamers.;Suzaan Marais.;David R Boulware.;Reinout van Crevel.;Rovina Ruslami.; .
来源: N Engl J Med. 2025年393卷24期2434-2446页
Tuberculous meningitis is often lethal, and many survivors have disabilities despite antimicrobial treatment and adjunctive glucocorticoid therapy. Standard-dose rifampin has limited central nervous system penetration. Whether high-dose rifampin could improve survival outcomes is unknown.
31. Cardiovascular Outcomes with Tirzepatide versus Dulaglutide in Type 2 Diabetes.
作者: Stephen J Nicholls.;Imre Pavo.;Deepak L Bhatt.;John B Buse.;Stefano Del Prato.;Steven E Kahn.;A Michael Lincoff.;Darren K McGuire.;Debra Miller.;Michael A Nauck.;Hiroshi Nishiyama.;Steven E Nissen.;Naveed Sattar.;Govinda Weerakkody.;Russell J Wiese.;Bernard Zinman.;Sophia Zoungas.;Jan Basile.;Melanie J Davies.;Francesco Giorgino.;Monika Kellerer.;Linong Ji.;Tamas Varkonyi.;Venu Menon.;Jonathan C Broder.;Alan Herschtal.;David D'Alessio.; .
来源: N Engl J Med. 2025年393卷24期2409-2420页
Tirzepatide, a dual incretin agonist of the glucagon-like peptide-1 and glucose-dependent insulinotropic polypeptide receptors, has favorable effects on glycemic control and body weight. The effects on cardiovascular outcomes are uncertain.
32. Universal Base-Edited CAR7 T Cells for T-Cell Acute Lymphoblastic Leukemia.
作者: Robert Chiesa.;Christos Georgiadis.;Hebatalla Rashed.;Roland Preece.;Prudence Hardefeldt.;Jan Chu.;Jemma Selvage.;Avijeet Mishra.;Batoul Ahmed.;Stuart Adams.;Rebecca Thomas.;Kimberly Gilmour.;Annie Etuk.;Deborah Yallop.;David O'Connor.;Waseem Qasim.; .
来源: N Engl J Med. 2026年394卷2期152-165页
CD7 is an attractive target for chimeric antigen receptor (CAR) T-cell therapy in relapsed or refractory T-cell acute lymphoblastic leukemia (ALL). Supportive results of first-in-human studies of base-edited anti-CD7 CAR (BE-CAR7) T cells with triple C→T deamination-mediated knockouts of TCRαβ, CD52, and CD7 have been reported previously.
33. Teclistamab plus Daratumumab in Relapsed or Refractory Multiple Myeloma.
作者: Luciano J Costa.;Nizar J Bahlis.;Aurore Perrot.;Ajay K Nooka.;Jin Lu.;Charlotte Pawlyn.;Roberto Mina.;Gaston Caeiro.;Alain Kentos.;Vania Hungria.;Donna Reece.;Ting Niu.;Anne K Mylin.;Charlotte T Hansen.;Raphael Teipel.;Britta Besemer.;Meletios A Dimopoulos.;Elena Zamagni.;Satoshi Yoshihara.;Kihyun Kim.;Chang Ki Min.;Paul Geerts.;Elena Van Leeuwen-Segarceanu.;Agata Tyczynska.;Juan Luis Reguera.;Magnus Johansson.;Markus Hansson.;Mehmet Turgut.;Mark Grey.;Surbhi Sidana.;Paula Rodriguez-Otero.;Joaquin Martinez-Lopez.;Hamza Hashmi.;Robin Carson.;Rachel Kobos.;Weili Sun.;Kristen Lantz.;Anne Seifert.;Deborah Briseno-Toomey.;Lisa O'Rourke.;Maria Rubin.;Diego Vieyra.;Lijuan Kang.;Maria Victoria Mateos.; .
来源: N Engl J Med. 2026年394卷8期739-752页
In a phase 1-2 trial, teclistamab, a bispecific antibody targeting CD3 on T-cell surfaces and B-cell maturation antigen on myeloma cells, showed durable responses in heavily pretreated patients with relapsed or refractory multiple myeloma. Daratumumab, a monoclonal antibody targeting CD38 protein, has shown survival benefit in patients with multiple myeloma.
34. Fixed-Duration versus Continuous Treatment for Chronic Lymphocytic Leukemia.
作者: Othman Al-Sawaf.;Janina Stumpf.;Can Zhang.;Florian Simon.;Francesc Bosch.;Emadoldin Feyzi.;Paolo Ghia.;Michael Gregor.;Arnon P Kater.;Vesa Lindström.;Mattias Mattsson.;Carsten U Niemann.;Philipp B Staber.;Tamar Tadmor.;Patrick Thornton.;Clemens-Martin Wendtner.;Ann Janssens.;Thomas Noesslinger.;Jan-Paul Bohn.;Caspar da Cunha-Bang.;Christian B Poulsen.;Juha Ranti.;Thomas Illmer.;Bjoern Schoettker.;Sebastian Böttcher.;Tobias Gaska.;Elisabeth Vandenberghe.;Ruth Clifford.;Ohad Benjamini.;Anna Maria Frustaci.;Lydia Scarfò.;Paolo Sportoletti.;John Schreurs.;Mark-David Levin.;Hanneke van der Straaten.;Marjolein van der Klift.;Hoa Tran.;Javier de la Serna.;Javier Loscertales.;Oscar Lindblad.;Anna Bergendahl Sandstedt.;Jeroen Goede.;Michael Baumann.;Anna Maria Fink.;Kirsten Fischer.;Matthias Ritgen.;Karl-Anton Kreuzer.;Christof Schneider.;Eugen Tausch.;Stephan Stilgenbauer.;Sandra Robrecht.;Barbara Eichhorst.;Michael Hallek.; .
来源: N Engl J Med. 2026年394卷11期1084-1096页
Treatment of chronic lymphocytic leukemia (CLL) currently consists of two main approaches - continuous therapy with Bruton's tyrosine kinase inhibitors and fixed-duration regimens combining venetoclax with either CD20 antibodies or Bruton's tyrosine kinase inhibitors. Comparisons of these two therapeutic approaches are lacking.
35. Prime Editing for p47phox-Deficient Chronic Granulomatous Disease.
作者: Jennifer L Gori.;Elie Haddad.;Haydar Frangoul.;Donald B Kohn.;Emma C Morris.;Bradley N Martin.;Briana A Deary.;McKinley Nickerson.;Rebecca L Scholz.;Isabel Fernandez.;Karine Leveille.;Suk See De Ravin.;Elizabeth M Kang.;Marie Pierzynski.;Tyra Estwick.;Patricia Littel.;Douglas B Kuhns.;Debra A Long Priel.;Pierre Teira.;Stuart Turvey.;Jordyn Arnold.;Misty D Evans.;Meghann McManus.;Ben Carpenter.;David P Waterman.;Andrew V Anzalone.;Alexandria Petrusich.;Christa E Osuna.;Tiernan T O'Malley.;Jacob Stewart-Ornstein.;Jack M Heath.;Barrett J Nehilla.;Mohammed Asmal.;Harry L Malech.
来源: N Engl J Med. 2026年394卷12期1195-1203页
Chronic granulomatous disease (CGD) is a severe monogenic immunodeficiency caused by damaging variants in genes required for microbicidal NADPH oxidase activity. Autosomal recessive p47phox-deficient CGD (p47-CGD) is predominantly caused by a two-nucleotide deletion in exon 2 (delGT) of NCF1. We developed PM359, an autologous CD34+ hematopoietic stem-cell therapy in which prime editing is used to correct delGT. Two participants received PM359 after myeloid conditioning with busulfan: neutrophils and platelets engrafted promptly in both patients. Adverse events were consistent with myeloid conditioning with busulfan. NADPH oxidase activity was observed in neutrophils within 1 month and was maintained for 6 months and 4 months as of the last follow-up visit in Participants 1 and 2, respectively. These results support further investigation of prime editing of CD34+ cells to treat p47-CGD. (Funded by Prime Medicine; ClinicalTrials.gov number, NCT06559176.).
36. Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
作者: Steven W Pipe.;Wolfgang Miesbach.;Michael Recht.;Frank W G Leebeek.;Nigel S Key.;Giancarlo Castaman.;Susan Lattimore.;Michiel Coppens.;Sandra Le Quellec.;Vaibhav Mahajan.;Sean Gill.;Douglass Drelich.;Paul E Monahan.; .
来源: N Engl J Med. 2026年394卷5期463-474页
Prophylactic treatment for hemophilia B necessitates lifelong, regular intravenous factor IX infusions. Gene therapy offers the possibility of a single-dose treatment that produces durable endogenous factor IX expression and disease control. Etranacogene dezaparvovec comprises an adeno-associated virus serotype 5 (AAV5) vector and the highly active Padua factor IX variant. The primary analysis of this study showed that etranacogene dezaparvovec reduced annualized bleeding rates and adverse events were mainly of low-grade severity. Final data from 5 years of follow-up are now available.
37. Dual Targeting of Extramedullary Myeloma with Talquetamab and Teclistamab.
作者: Shaji Kumar.;María-Victoria Mateos.;Jing Christine Ye.;Shebli Atrash.;Hila Magen.;Hang Quach.;Michael P Chu.;Suzanne Trudel.;Joshua Richter.;Paula Rodríguez-Otero.;Hun Chuah.;Moshe Gatt.;Eva Medvedova.;Shahzad Raza.;Dok Hyun Yoon.;Tadao Ishida.;Jeffrey V Matous.;Laura Rosiñol.;Koichi Onodera.;Emma Scott.;Christoph Heuck.;Jenny Zhang.;Todd Henninger.;Lisa O'Rourke.;Payal Thakkar.;Mariacristina Festa.;Lin Huang.;Jiangxiu Zhou.;Mikihiro Takamoto.;Lixia Pei.;Jiashen Lu.;Nicholas Au.;Maria Krevvata.;Saad Z Usmani.;Yael C Cohen.; .
来源: N Engl J Med. 2026年394卷1期51-61页
Patients with plasmacytomas that are noncontiguous with bone marrow (true extramedullary myeloma) are at high risk for disease progression or relapse. Phase 1 of the RedirecTT-1 study showed promising efficacy with dual-antigen targeting of myeloma with talquetamab (anti-G protein-coupled receptor family C group 5 member D) plus teclistamab (anti-B-cell maturation antigen) in patients with triple-class-exposed relapsed or refractory multiple myeloma, including those with true extramedullary myeloma.
38. Noninferiority of One HPV Vaccine Dose to Two Doses.
作者: Aimée R Kreimer.;Carolina Porras.;Danping Liu.;Allan Hildesheim.;Loretto J Carvajal.;Rebeca Ocampo.;Byron Romero.;Mitchell H Gail.;Bernal Cortes.;Monica S Sierra.;Karla Coronado.;Joshua Sampson.;Carolina Coto.;Casey L Dagnall.;Daniela Mora.;Troy J Kemp.;Michael Zuniga.;Ligia A Pinto.;Gloriana Barrientos.;John Schussler.;Yenory Estrada.;Cristian Montero.;Carlos Avila.;Dave Ruggieri.;Jean T Cyr.;Stephen Chanock.;Douglas R Lowy.;John T Schiller.;Rolando Herrero.
来源: N Engl J Med. 2025年393卷24期2421-2433页
Multidose human papillomavirus (HPV) vaccination is efficacious, yet the vaccine has been underused globally. Emerging data suggest that a single dose may provide protection. Whether a single dose of HPV vaccine would provide similar protection to two doses is uncertain.
39. Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN.
作者: Fadi Fakhouri.;Andrew S Bomback.;Gema Ariceta.;Yahsou Delmas.;Bradley P Dixon.;Daniel P Gale.;Larry A Greenbaum.;Seung Hyeok Han.;Nicole Isbel.;Moglie Le Quintrec.;Christoph Licht.;Antonio Mastrangelo.;Masashi Mizuno.;Maria Izabel Neves de Holanda.;Matthew C Pickering.;Giuseppe Remuzzi.;Nicole Van De Kar.;Marina Vivarelli.;Patrick D Walker.;Dean Wallace.;Daniel Zecher.;Cedric Francois.;Pascal Deschatelets.;Li Li.;Zhongshen Wang.;Lydia Abad-Franch.;Nils Kinnman.;Luis López-Lázaro.;Johan Szamosi.;Carla M Nester.; .
来源: N Engl J Med. 2025年393卷22期2210-2220页
C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis (MPGN) generally result in glomerular C3 deposition and irreversible kidney damage. The efficacy and safety of pegcetacoplan, a C3 and C3b inhibitor, in persons with C3 glomerulopathy or primary immune-complex MPGN are unclear.
40. Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia.
作者: Hanny Al-Samkari.;Josefien Hessels.;Antoni Riera-Mestre.;Sophie Dupuis-Girod.;Thibaut Van Zele.;Vicente Gómez Del Olmo.;Pamela G Hodges.;Raquel Torres-Iglesias.;Roberto Bertè.;Pierre Saint-Mezard.;Hedvika Lazar.;Nicholas Benedict.;Debra Barker.;Corrado Bernasconi.;Damien Picard.;Elisabetta Buscarini.;Hans-Jurgen Mager.
来源: N Engl J Med. 2025年393卷21期2131-2141页
Hereditary hemorrhagic telangiectasia (HHT) can cause recurrent, severe epistaxis, as well as anemia and reduced quality of life. The disease remains without licensed therapies worldwide.
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