What compassionate use means for gene therapies

被引：17

作者：

Chapman, Carolyn Riley ^{[1
]}

Moch, Kenneth I. ^{[2
]}

McFadyen, Andrew ^{[3
]}

Kearns, Lisa ^{[1
]}

Watson, Tom ^{[4
]}

Furlong, Pat ^{[5
]}

Bateman-House, Alison ^{[1
]}

机构：

[1] NYU, Sch Med, Dept Populat Hlth, Div Med Eth, New York, NY 10016 USA

[2] Cognit Therapeut, Pittsburgh, PA USA

[3] Isaac Fdn, Campbellford, ON, Canada

[4] Bion Early Access Programs, Willington, England

[5] PPMD, Hackensack, NJ USA

来源：

NATURE BIOTECHNOLOGY | 2019年 / 37卷 / 04期

关键词：

D O I：

10.1038/s41587-019-0081-7

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

引用

页码：352 / 355

页数：4

共 24 条

[1]

[Anonymous], 2018, CNBC

[2]

[Anonymous], 2017, FOCUS PROJ FIN CUR U

[3]

[Anonymous], 2018, MIT TECHNOLOGY REV

[4]

ASCO, 2017, ASCO FRIENDS CANC RE

[5] Ethics review in compassionate use [J].

Borysowski, Jan ;

Ehni, Hans-Jorg ;

Gorski, Andrzej .

BMC MEDICINE, 2017, 15

[6]

Brau R., 2018, LIFE SCIENCE LEADER

[7]

Caplan A, 2014, HLTH AFFAIRS BLOG, DOI [10.1377/hblog20140827.041027/full, DOI 10.1377/HBLOG20140827.041027/FULL/]

[8] Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials [J].

Caplan, Arthur L. ;

Teagarden, J. Russell ;

Kearns, Lisa ;

Bateman-House, Alison S. ;

Mitchell, Edith ;

Arawi, Thalia ;

Upshur, Ross ;

Singh, Ilina ;

Rozynska, Joanna ;

Cwik, Valerie ;

Gardner, Sharon L. .

JOURNAL OF MEDICAL ETHICS, 2018, 44 (11) :761-767

[9]

Chapman C. R., 2017, HLTH AFFAIRS BLOG, DOI [10.1377/hblog20171129.293917/full/, DOI 10.1377/HBLOG20171129.293917/FULL/]

[10] Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety [J].

Cicalese, Maria Pia ;

Ferrua, Francesca ;

Castagnaro, Laura ;

Rolfe, Katie ;

De Boever, Erika ;

Reinhardt, Rickey R. ;

Appleby, Jonathan ;

Roncarolo, Maria Grazia ;

Aiuti, Alessandro .

MOLECULAR THERAPY, 2018, 26 (03) :917-931

← 1 2 3 →