Faculty Publications

Fulfilling the Mandate of the US Food and Drug Administration's Accelerated Approval Pathway: The Need for Reforms.

Journal:

JAMA Intern Med

181(10):1275-1276

Publish Date:

2021-10-01

All Authors:

Gyawali B, Ross JS, Kesselheim AS.

Author Profiles:

Aaron Seth Kesselheim

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Tweetorials for Medical Educators.

Journal:

J Grad Med Educ

13(5):723-725

Publish Date:

2021-10-01

All Authors:

Breu AC, Abrams HR, Manning KD, Cooper AZ.

Author Profiles:

Anthony Charles Breu

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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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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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