Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project-Reference-Cited by-同舟云学术

Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project

Published:2018-05-20 Issue:4 Volume:24 Page:e167-e172
ISSN:1351-8216
Container-title:Haemophilia
language:en
Short-container-title:Haemophilia

Author:

Iorio A.¹^ORCID,Skinner M. W.²³,Clearfield E.⁴,Messner D.⁴,Pierce G. F.⁵^ORCID,Witkop M.²,Tunis S.⁴,

Affiliation:

1. Department of Health Research Methods, Evidence and Impact; Department of Medicine; McMaster University; Hamilton ON Canada

2. National Hemophilia Foundation; New York NY USA

3. Institute for Policy Advancement, Ltd; Washington DC USA

4. Center for Medical Technology Policy; Baltimore MD USA

5. Third Rock Ventures; San Francisco CA USA

Funder

Pfizer

Spark Therapeutics

National Hemophilia Foundation

Shire

St. Jude Children's Research Hospital

Bayer

BioMarin Pharmaceutical

UniQure

Publisher

Wiley

Subject

Genetics(clinical),Hematology,General Medicine

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1111/hae.13504/fullpdf

Reference15 articles.

1. Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B;Iorio;Cochrane Database Syst Rev,2011

2. Emicizumab prophylaxis in hemophilia A with inhibitors;Oldenburg;N Engl J Med,2017

3. The cost of severe haemophilia in Europe: the CHESS study;O'Hara;Orphanet J Rare Dis,2017

4. Association between physical activity and risk of bleeding in children with hemophilia;Broderick;JAMA,2012

5. AAV5-factor VIII gene transfer in severe hemophilia A;Rangarajan;N Engl J Med,2017

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