Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria-Reference-Cited by-同舟云学术

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

Published:2020-06-11 Issue:24 Volume:382 Page:2289-2301
ISSN:0028-4793
Container-title:New England Journal of Medicine
language:en
Short-container-title:N Engl J Med

Author:

Balwani Manisha¹,Sardh Eliane¹,Ventura Paolo¹,Peiró Paula Aguilera¹,Rees David C.¹,Stölzel Ulrich¹,Bissell D. Montgomery¹,Bonkovsky Herbert L.¹,Windyga Jerzy¹,Anderson Karl E.¹,Parker Charles¹,Silver Samuel M.¹,Keel Siobán B.¹,Wang Jiaan-Der¹,Stein Penelope E.¹,Harper Pauline¹,Vassiliou Daphne¹,Wang Bruce¹,Phillips John¹,Ivanova Aneta¹,Langendonk Janneke G.¹,Kauppinen Raili¹,Minder Elisabeth¹,Horie Yutaka¹,Penz Craig¹,Chen Jihong¹,Liu Shangbin¹,Ko John J.¹,Sweetser Marianne T.¹,Garg Pushkal¹,Vaishnaw Akshay¹,Kim Jae B.¹,Simon Amy R.¹,Gouya Laurent¹

Affiliation:

1. From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King’s...

Funder

Alnylam Pharmaceuticals

Publisher

Massachusetts Medical Society

Subject

General Medicine

Link

http://www.nejm.org/doi/pdf/10.1056/NEJMoa1913147

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