Policy for rare diseases

Professor Bobby Gaspar is a distinguished physician-scientist who is a thought leader in translating basic research from bench-to-bedside and strategic work that facilitated bringing life-saving therapies to patients with rare diseases. He has over 30 years of experience in pediatric medicine workin...

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Detalles Bibliográficos
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Q&A
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9974947/
https://www.ncbi.nlm.nih.gov/pubmed/36854888
http://dx.doi.org/10.1038/s43856-023-00254-4
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description Professor Bobby Gaspar is a distinguished physician-scientist who is a thought leader in translating basic research from bench-to-bedside and strategic work that facilitated bringing life-saving therapies to patients with rare diseases. He has over 30 years of experience in pediatric medicine working in the NHS and the biotechnology sector, and is the founding member of Orchard Therapeutics, where he serves as Chief Executive Officer. In this Q&A, Professor Gaspar provides insight into the regulatory approval and policy considerations for bringing novel therapies for rare diseases from discovery through to clinical application.
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spelling pubmed-99749472023-03-02 Policy for rare diseases Commun Med (Lond) Q&A Professor Bobby Gaspar is a distinguished physician-scientist who is a thought leader in translating basic research from bench-to-bedside and strategic work that facilitated bringing life-saving therapies to patients with rare diseases. He has over 30 years of experience in pediatric medicine working in the NHS and the biotechnology sector, and is the founding member of Orchard Therapeutics, where he serves as Chief Executive Officer. In this Q&A, Professor Gaspar provides insight into the regulatory approval and policy considerations for bringing novel therapies for rare diseases from discovery through to clinical application. Nature Publishing Group UK 2023-02-28 /pmc/articles/PMC9974947/ /pubmed/36854888 http://dx.doi.org/10.1038/s43856-023-00254-4 Text en © Springer Nature Limited 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Q&A
Policy for rare diseases
title Policy for rare diseases
title_full Policy for rare diseases
title_fullStr Policy for rare diseases
title_full_unstemmed Policy for rare diseases
title_short Policy for rare diseases
title_sort policy for rare diseases
topic Q&A
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9974947/
https://www.ncbi.nlm.nih.gov/pubmed/36854888
http://dx.doi.org/10.1038/s43856-023-00254-4

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