Peri-transfusion quality-of-life assessment for patients with myelodysplastic syndromes.

Journal:
Transfusion
61(10):2830-2836
Publish Date:
2021-10-01
All Authors:
Abel GA, Klepin HD, Magnavita ES, Jaung T, Lu W, Shallis RM, Hantel A, Bahl NE, Dellinger-Johnson R, Winer ES, Zeidan AM.
Author Profiles:
Gregory Abel

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DBS and Autonomy: Clarifying the Role of Theoretical Neuroethics.

Journal:
Neuroethics
14(Suppl 1):83-93
Publish Date:
2021-10-01
All Authors:
Zuk P, Lázaro-Muñoz G.
Author Profiles:

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Evidence generation and reproducibility in cell and gene therapy research: A call to action.

Journal:
Mol Ther Methods Clin Dev
22:11-14
Publish Date:
2021-09-10
All Authors:
Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA.
Author Profiles:

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Regulatory and clinical consequences of negative confirmatory trials of accelerated approval cancer drugs: retrospective observational study.

Journal:
BMJ
374:n1959
Publish Date:
2021-09-08
All Authors:
Gyawali B, Rome BN, Kesselheim AS.
Author Profiles:
Aaron Seth Kesselheim

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Gene therapy in the putamen for curing AADC deficiency and Parkinson's disease.

Journal:
EMBO Mol Med
13(9):e14712
Publish Date:
2021-09-07
All Authors:
Hwu PW, Kiening K, Anselm I, Compton DR, Nakajima T, Opladen T, Pearl PL, Roubertie A, Roujeau T, Muramatsu SI.
Author Profiles:

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TNPO2 variants associate with human developmental delays, neurologic deficits, and dysmorphic features and alter TNPO2 activity in Drosophila.

Journal:
Am J Hum Genet
108(9):1669-1691
Publish Date:
2021-09-02
All Authors:
Goodman LD, Cope H, Nil Z, Ravenscroft TA, Charng WL, Lu S, Tien AC, Pfundt R, Koolen DA, Haaxma CA, Veenstra-Knol HE, Wassink-Ruiter JSK, Wevers MR, Jones M, Walsh LE, Klee VH, Theunis M, Legius E, Steel D, Barwick KES, Kurian MA, Mohammad SS, Dale RC, Terhal PA, van Binsbergen E, Kirmse B, Robinette B, Cogné B, Isidor B, Grebe TA, Kulch P, Hainline BE, Sapp K, Morava E, Klee EW, Macke EL, Trapane P, Spencer C, Si Y, Begtrup A, Moulton MJ, Dutta D, Kanca O, Wangler MF, Yamamoto S, Bellen HJ, Tan QK.
Author Profiles:
Lauren Walsh

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How Should Clinicians and Organizations Assess Risks and Benefits of First-in-Human Implantation of Investigational Devices?

Journal:
AMA J Ethics
23(9):E673-678
Publish Date:
2021-09-01
All Authors:
Brown BL, Kesselheim AS.
Author Profiles:
Aaron Seth Kesselheim

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Analysis of Launch and Postapproval Cancer Drug Pricing, Clinical Benefit, and Policy Implications in the US and Europe.

Journal:
JAMA Oncol
7(9):e212026
Publish Date:
2021-09-01
All Authors:
Vokinger KN, Hwang TJ, Daniore P, Lee CC, Tibau A, Grischott T, Rosemann TJ, Kesselheim AS.
Author Profiles:
Aaron Seth Kesselheim

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Oncogenic switch and single-agent MET inhibitor sensitivity in a subset of EGFR-mutant lung cancer.

Journal:
Sci Transl Med
13(609):eabb3738
Publish Date:
2021-09-01
All Authors:
Eser PÖ, Paranal RM, Son J, Ivanova E, Kuang Y, Haikala HM, To C, Okoro JJ, Dholakia KH, Choi J, Eum Y, Ogino A, Missios P, Ercan D, Xu M, Poitras MJ, Wang S, Ngo K, Dills M, Yanagita M, Lopez T, Lin M, Tsai J, Floch N, Chambers ES, Heng J, Anjum R, Santucci AD, Michael K, Schuller AG, Cross D, Smith PD, Oxnard GR, Barbie DA, Sholl LM, Bahcall M, Palakurthi S, Gokhale PC, Paweletz CP, Daley GQ, Jänne PA.
Author Profiles:

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International reference pricing for prescription drugs: a landscape analysis.

Journal:
J Manag Care Spec Pharm
27(9):1309-1313
Publish Date:
2021-09-01
All Authors:
Rand LZ, Kesselheim AS.
Author Profiles:
Leah Rand

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