Risk-benefit assessment for Gene Therapy in Haemophilia ft. Declan Noone, EHC President
Haemcast20 Des 2021

Risk-benefit assessment for Gene Therapy in Haemophilia ft. Declan Noone, EHC President

Declan Noone, President of the European Haemophilia Consortium (EHC) joins Haemcast hosts, Dr Kate Khair and Luke Pembroke to discuss the complexities surrounding risk-benefit assessment when making a decision around gene therapy. From considering the patients' and healthcare professional perspectives to the regulators and payers, thorough risk-benefit assessment processes will be required in order to make informed decisions about gene therapy for haemophilia. But what is needed from the community to support these decision making processes? How can we ensure equal access and opportunity to gene therapy in the future? How do we begin to try quantifying the benefits of gene therapy without focusing solely on annual bleed rates and factor levels? Listen in to hear the thoughts of our experts in this final episode of Haemcast for 2021.

Thank you to all of our listeners throughout the year for tuning in and supporting Haemcast. The feedback we have received has been amazing and confirmed that it serves as a valuable resource for the community. If you want to help supporting us, please do leave us a review and rating on the platform you listen on e.g. Apple podcasts, Spotify and be sure to share Haemcast with your colleagues and friends.

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Episoder(38)

Reflections on a life with severe haemophilia. Part 2: Education and dreams of the future

Reflections on a life with severe haemophilia. Part 2: Education and dreams of the future

We rejoin Terence O'Rourke to as he reflects on growing up with haemophilia, not being able to go to school, his education, and the impact of this on his life. Now in his 80s, Terence also looks back ...

30 Apr 202513min

Reflections on a life with severe haemophilia: In conversation with Terence O ’ Rourke

Reflections on a life with severe haemophilia: In conversation with Terence O ’ Rourke

In the first part of a six-part mini-series, Terence O’Rourke looks back on his childhood memories of living with severe haemophilia, including his education and experience of treatment. Terence, who ...

17 Apr 202517min

In conversation with Kate Khair: reflecting on a lifetime's achievement in haemophilia and bleeding disorders

In conversation with Kate Khair: reflecting on a lifetime's achievement in haemophilia and bleeding disorders

In this episode of Haemcast, we speak to our very own Dr Kate Khair following her Lifetime Achievement award at EAHAD 2024. Tune in to hear about Kate's journet into nursing, the first patient she me...

20 Mar 202443min

Pathway to Cures; the venture philanthropy fund focused on transformative treatments for inheritable blood disorders ft. Len Valentino and Teri Willey

Pathway to Cures; the venture philanthropy fund focused on transformative treatments for inheritable blood disorders ft. Len Valentino and Teri Willey

In this episode, host Luke Pembroke (Director of Community Engagement, Haemnet) sat down with Len Valentino (P2C Chief Executive Officer; and NBDF President & CEO) and Teri Willey (P2C Managing Direct...

9 Jan 202434min

"Keep speaking their names. Keep remembering them." - Commemorating World AIDS Day 2023 ft. Mark Ward, LGBT Ambassador,The Haemophilia Society UK

"Keep speaking their names. Keep remembering them." - Commemorating World AIDS Day 2023 ft. Mark Ward, LGBT Ambassador,The Haemophilia Society UK

In this episode we commemorate World AIDS Day 2023. Host, Dr Kate Khair is joined by contaminated blood scandal campaigner and The Haemophilia Society UK's LGBT Ambassador to discuss the impact HIV an...

1 Des 202339min

Von Willebrand what?... Disease? Disorder? Dismissed? - featuring community advocates Sunny Maini & Hannah Yarnall, The UK Haemophilia Society

Von Willebrand what?... Disease? Disorder? Dismissed? - featuring community advocates Sunny Maini & Hannah Yarnall, The UK Haemophilia Society

In this episode, Director of Community Engagement, Luke Pembroke, and Director of Research, Kate Khair spoke with community advocates Sunny Maini and Hannah Yarnalll. Sunny and Hannah share their exp...

9 Aug 202344min

Psychologist Sylvia von Mackensen shares how her leap from sick buildings led her to become the guru of quality of life in haemophilia

Psychologist Sylvia von Mackensen shares how her leap from sick buildings led her to become the guru of quality of life in haemophilia

From the development of standardised tools, their benefits and limitations, through to the challenges and changes we face in assessing quality of life for people with haemophilia and bleeding disorder...

6 Jul 202331min

The Qualitative Revolution has Begun | Creative Qualitative Research feat. Dr Rich Gorman, Social Scientist at at Brighton and Sussex Medical School

The Qualitative Revolution has Begun | Creative Qualitative Research feat. Dr Rich Gorman, Social Scientist at at Brighton and Sussex Medical School

Qualitative research is increasingly challenged to think creatively and critically about how accounts of lived experience might be collected, curated, and shared. Historically it could be said that qu...

13 Apr 202344min

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