Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH-Reference-Cited by-同舟云学术

Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH

Published:2020-10-29 Issue:11 Volume:18 Page:3074-3077
ISSN:1538-7933
Container-title:Journal of Thrombosis and Haemostasis
language:en
Short-container-title:J. Thromb. Haemost.

Author:

Konkle Barbara¹^ORCID,Pierce Glen²^ORCID,Coffin Donna²^ORCID,Naccache Mayss²,Clark R. Cary³,George Lindsey⁴^ORCID,Iorio Alfonso⁵^ORCID,O’Mahony Brian⁶^ORCID,Pipe Steven⁷^ORCID,Skinner Mark⁸^ORCID,Watson Crystal⁹,Peyvandi Flora¹⁰^ORCID,Mahlangu Johnny¹¹^ORCID,

Affiliation:

1. Bloodworks Northwest, Research Institute University of Washington Seattle WA USA

2. World Federation of Hemophilia Montreal QC USA

3. ISTH Carrboro NC USA

4. The Children's Hospital of Philadelphia Philadelphia PA USA

5. Clinical Epidemiology and Biostatistics McMaster University Hamilton ON Canada

6. Irish Haemophilia Society Dublin Ireland

7. Pediatrics University of Michigan Ann Arbor MI USA

8. Institute for Policy Advancement Ltd WashingtonDCUSA

9. American Thrombosis and Hemostasis Network New York NY USA

10. Internal Medicine Faculty of Medicine and Surgery University of Milan Milan Italy

11. Faculty of Health Sciences and NHLS University of the Witwatersrand Johannesburg South Africa

Publisher

Wiley

Subject

Hematology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/jth.15023

Reference19 articles.

1. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia

2. A Molecular Revolution in the Treatment of Hemophilia

3. Gene therapy for hemophilia

4. Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

5. Minimal dataset for post-registration surveillance of new drugs in hemophilia: communication from the SSC of the ISTH

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