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Wednesday 27th April 2022, 15:30-16:30 BST Prof. Laurent Servais, University of Oxford
Three diseases modifying therapies are now approved for use by NICE, but a huge burden remains for family and patients. Indeed, SMA remains associated with a severe disability in patients diagnosed after developing symptoms. What are the next steps? Could the association of drugs provide an additional benefit? Is there any other target that could be considered? Finally, could newborn screening constitute a valuable solution to decrease patient and societal burden in the long term?
A step-by-step guide to setting up and running a research-grade vector production facility within an academic institute: challenges to face, manufacturing platforms and business models for a successful vector manufacturing laboratory.
Wednesday 23rd February 2022, 14:00-15:00 GMT Dr. Giulia Massaro, University College London
Monday 20th September 2021, 14:00-15:00 BST Prof. Luigi Naldini, Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.
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Vered Smith is our 2020 Science Writing Competition winner with her article about glaucoma.