Note: This webinar took place on 29 October 2018. You can find a video recording and the presentations used during the webinar below. Webinar introduction Amongst other profound changes and consequences,...
The starter kits provide you with links to relevant background reading in the toolbox and associated PowerPoint slide decks and media in order to prepare a single or multi-day...
Introduction A collaboration of Genzyme, International Pompe Association (IPA); UK, Dutch, and US Pompe patient organisations, Erasmus Medical Center and Duke University to establish appropriate measures to ensure the...
Vaccines are biological preparations developed from modified versions of the micro-organisms that cause a disease. Vaccines improve or convey immunity to particular diseases, usually in an effort to prevent...
Once a target receptor molecule or an enzyme has been identified, scientists begin to look for potential compounds that will interact with the target to correct disease-related activity.These molecules...
What is compassionate use? The term ‘compassionate use’ refers to special programmes set up to make unauthorised medicines available to patients. In particular, this may be a way of...
Transcription My name is Karen Facey. I am a statistician by training. I’ve worked in drug development, medicines regulation and health technology assessment. Since being involved in health...
Phase II clinical studies, or proof of concept studies, begin to test the effect of a medicine on the target disease in humans. These studies are usually run as...
Diagnosed with MS and then …. When I got my diagnosis in 2005, I didn’t know a lot about multiple sclerosis. I was absolutely uninformed. Why should we think...
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