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Fraud, Abuse, and Financial Conflicts of Interest
Zack Buck, J.D., M.B.E.
doi : 10.1056/NEJMp2201628
Protecting the Legitimacy of Medical Expertise
Richard J. Baron, M.D., and Carl H. Coleman, J.D.
doi : 10.1056/NEJMp2214120
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.
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.
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.
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.
Acute Kidney Injury in Patients with Cirrhosis
Mitra K. Nadim, M.D., and Guadalupe Garcia-Tsao, M.D.
doi : 10.1056/NEJMra2215289
Pseudothrombocytosis
Konstantinos Liapis, M.D., Ph.D., and Stavros Papadakis, M.D.
doi : 10.1056/NEJMicm2209299
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
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
Focused Ultrasound Ablation of the Globus Pallidus Internus for Parkinson’s Disease
Anette Schrag, Ph.D., F.R.C.P.
doi : 10.1056/NEJMe2215506
Hemophilia A Gene Therapy — Some Answers, More Questions
Lindsey A. George, M.D.
doi : 10.1056/NEJMe2212347
