New England Journal of Medicine




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سفارش

Fraud, Abuse, and Financial Conflicts of Interest

Zack Buck, J.D., M.B.E.

doi : 10.1056/NEJMp2201628

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Protecting the Legitimacy of Medical Expertise

Richard J. Baron, M.D., and Carl H. Coleman, J.D.

doi : 10.1056/NEJMp2214120

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The Princess and the He

Brendan M. Reilly, M.D.

doi : 10.1056/NEJMp2215941

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Trial of Globus Pallidus Focused Ultrasound Ablation in Parkinson’s Disease

Vibhor Krishna, M.D., Paul S. Fishman, M.D., Ph.D., Howard M. Eisenberg, M.D., Michael Kaplitt, M.D., Ph.D., Gordon Baltuch, M.D., Jin Woo Chang, M.D., Wei-Chieh Chang, M.D., Raul Martinez Fernandez, M.D., Marta del Alamo, M.D., Casey H. Halpern, M.D., Pejman Ghanouni, M.D., Ph.D., Roberto Eleopra, M.D., Ph.D.,

doi : 10.1056/NEJMoa2202721

Unilateral focused ultrasound ablation of the internal segment of globus pallidus has reduced motor symptoms of Parkinson’s disease in open-label studies.

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Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Johnny Mahlangu, M.B., B.Ch., M.Med., Radoslaw Kaczmarek, Ph.D., Annette von Drygalski, M.D., Pharm.D., Susan Shapiro, M.D., Ph.D., Sheng-Chieh Chou, M.D., Margareth C. Ozelo, M.D., Ph.D., Gili Kenet, M.D., Flora Peyvandi, M.D., Ph.D., Michael Wang, M.D., Bella Madan, M.D., Nigel S. Key, M.D., Michael Laffan, D.M., Ph.D.,

doi : 10.1056/NEJMoa2211075

Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a phase 3 study of the efficacy and safety of valoctocogene roxaparvovec therapy evaluated after 52 weeks in men with severe hemophilia A have been published previously.

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Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Steven W. Pipe, M.D., Frank W.G. Leebeek, M.D., Ph.D., Michael Recht, M.D., Ph.D., Nigel S. Key, M.B., Ch.B., Giancarlo Castaman, M.D., Wolfgang Miesbach, M.D., Susan Lattimore, R.N., Kathelijne Peerlinck, M.D., Ph.D., Paul Van der Valk, M.D., Michiel Coppens, M.D., Ph.D., Peter Kampmann, M.D., Karina Meijer, M.D., Ph.D.,

doi : 10.1056/NEJMoa2211644

Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding without burdensome factor IX replacement.

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Rucaparib or Physician’s Choice in Metastatic Prostate Cancer

Karim Fizazi, M.D., Ph.D., Josep M. Piulats, M.D., Ph.D., M. Neil Reaume, M.D., Peter Ostler, M.D., Ray McDermott, M.B., B.Ch., B.A.O., Ph.D., Joel R. Gingerich, M.D., Elias Pintus, M.D., Srikala S. Sridhar, M.D., Richard M. Bambury, M.D., Urban Emmenegger, M.D., Henriette Lindberg, M.D., Ph.D., David Morris, M.D.,

doi : 10.1056/NEJMoa2214676

In a phase 2 study, rucaparib, an inhibitor of poly(ADP-ribose) polymerase (PARP), showed a high level of activity in patients who had metastatic, castration-resistant prostate cancer associated with a deleterious BRCA alteration. Data are needed to confirm and expand on the findings of the phase 2 study.

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Acute Kidney Injury in Patients with Cirrhosis

Mitra K. Nadim, M.D., and Guadalupe Garcia-Tsao, M.D.

doi : 10.1056/NEJMra2215289

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Pseudothrombocytosis

Konstantinos Liapis, M.D., Ph.D., and Stavros Papadakis, M.D.

doi : 10.1056/NEJMicm2209299

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Patent Urachus with Posterior Urethral Valves

Karen L. Petersen, M.B., B.Ch., M.Med., and Derek Harrison, M.B., Ch.B., M.Med.

doi : 10.1056/NEJMicm2204739

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Case 6-2023: A 68-Year-Old Man with Recurrent Strokes

Aneesh B. Singhal, M.D., M.B., B.S., Scott B. Silverman, M.D., Javier M. Romero, M.D., and Melanie Lang-Orsini, M.D.

doi : 10.1056/NEJMcpc2211368

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Focused Ultrasound Ablation of the Globus Pallidus Internus for Parkinson’s Disease

Anette Schrag, Ph.D., F.R.C.P.

doi : 10.1056/NEJMe2215506

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Hemophilia A Gene Therapy — Some Answers, More Questions

Lindsey A. George, M.D.

doi : 10.1056/NEJMe2212347

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Effectiveness of Bivalent Boosters against Severe Omicron Infection

doi : 10.1056/NEJMc2215471

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Safety and Efficacy of Denosumab for Fibrous Dysplasia of Bone

doi : 10.1056/NEJMc2214862

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Psilocybin for Treatment-Resistant Depression

doi : 10.1056/NEJMc2215459

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