World Symposium day 3- clinical trials for rare diseases

It’s day 3 of the World symposium. A meeting with mostly 15 min presentations running from 8am to 4.30pm with breaks and lunch. It runs like clockwork and virtually every talk had good questions. Yet the most annoying thing I can say about the meeting is that they excessively use music and cut off the sound to speakers after a set time. Now many would say it is a sensible way to keep things moving along. But in many cases answers to important questions were unheard because some power wielding audio visual person decided they would cut off the sound. This in a way is censorship. I think it’s wrong to remove the decision making of the chairs of the scientific session and hand to a non scientist. My only other quibble with the meeting is the strong exclusion of use of mobile phones to take photographs of posters. I have no problem with this during talks. This seems a little draconian because most people do not have print outs of posters and many also had QR codes on their posters to enable people to use phones to download a PDF.

Alright I am off my soapbox. The meeting is also fascinating because of mix preclinical and clinical presentations, the blending of academics and biotechs, the attendance and participation of patients, advocates and scientists. It’s very eye opening in that respect. I think it’s rare to have a meeting in which there is this connection between the basic scientist all the way to the patient.

What could other meetings learn from this? Bringing together so many stakeholders certainly keeps it interesting and really I think focuses the research presented. Many of the clinical presentations made a point either before or after their talks of thanking the patients. Several of the talks also used video so you could see the clinical differences in different aspects of the rare disease.

Today was the turn of clinical trials to be highlighted such as Pompe, Hurler, Batten, Fabry, Gaucher, Niemann-Pick, Wolman disease. Most described enzyme replacement with occasional discussion if chaperones such as migalastat for Fabry and the substrate inhibitor Eliglustat for Gaucher. So many different diseases, a great deal of technical language to decipher, and a huge amount (if not a overwhelming) information.


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