Year: 2020
Language: English
Author(s): World Federation of Hemophilia
In this session from the WFH 2020 Virtual Summit, Steve Pipe and Luigi Naldini explore the promise of gene therapy and the broad-based multi-stakeholder preparation that is needed to deliver on this achievement.
Steve Pipe explores that, although we currently stand on the threshold of this achievement, delivering on this promise will require broad-based multi-stakeholder preparation with a focused emphasis on education, approval of safe and effective therapies, removal of barriers to access and excellence in clinical delivery.
Luigi Naldini continues to investigate new strategies to overcome the major hurdles to safe and effective gene transfer and translate them into new therapeutic strategies for genetic disease and cancer. Naldini presents his data regarding the further development of lentiviral vectors as a systemic gene delivery vehicle to complement the reach of AAV vectors.
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The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.
The WFH does not promote any particular pharmaceutical product and any mention of any commercial brand in this presentation is strictly for educational purposes.
This document is an introductory guide for people with hemophilia who wish to join the World Federation of Hemophilia (WFH)
This document is an introductory guide for people with hemophilia who wish to join the World Federation of Hemophilia (WFH)
The World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR) welcomes the participation of all Hemophilia Treatment Centres (HTCs) involved
The World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR) welcomes the participation of all Hemophilia Treatment Centres (HTCs) involved
This document is an introductory guide to the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). It answers basic
This document is an introductory guide to the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). It answers basic
