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CSAC Update July 2024


Hear from our trustee Dr Fiona Stewart as she reflects on a busy past six months of CSAC (Clinical Scientific Advisory Committee) achievements and looks ahead to an exciting autumn.

New members, more experience

It's been a busy few months for CSAC. We are delighted to now have Professor Simon Jones and Dr David Whiteman on CSAC. Simon is a Professor of paediatrics in Manchester and has a huge amount of clinical and research expertise. David has spent a lifetime in the pharma industry and has a wealth of experience in drug trials, particularly in the area of neuronopathic diseases. His insights are extremely valuable and are very much welcomed.

I think we are very fortunate to have such depth and breadth of experience on CSAC.

On the road

In February, I attended the WORLD symposium in San Diego together with other CSAC members, Sophie Thomas, our CEO Bob Stevens, as well as Bennie and Alex from RDRP. I was able to see how hard the team worked and how many interactions they had with everyone including other patient organisations, government bodies and pharma companies.

It was also great to see that CSAC, RDRP and the MPS Society are held in such high regard.

In March, CSAC member Prof Derralynn Hughes played a major role in planning and contributing to the very successful Fabry Matters conference.

In April, I went with Bob and Sophie to Würzburg to attend the 17th International MPS Symposium. Prior to the main conference there were two pre-meetings. The first provided an insight on the use of surrogate biomarkers in drug trials and was organised by Simon Jones and Prof Joe Muenzer. The second meeting focused on difficult airways and was spearheaded by Simon Jones and Sophie Thomas.

I felt both of these meetings were extremely productive and should lead to better outcomes for our families both in terms of research trials and clinical management.

Sophie is currently collating all the discussions from the respiratory meeting which is quite a job! The actual Symposium was very good too and we welcomed opportunities to interact with pharma and hear about possible new trials.


CSAC put out a single call for grants this year, so we could assess all applications together. Overall we had six applications and are in the process of taking three of them forward for further consideration. We have less money to award than in previous years, so we need to make sure we fund projects that are of most benefit to members of the MPS Society.

Going forward, we are planning another CSAC Open Day in the autumn where we are hoping to showcase some of the work which we have funded.

We really appreciate the fact that the money raised by our members helps fund the grants we award, to then ultimately benefit our members and their families. Please continue to do the great work you are doing.

We will be announcing the date of our CSAC open day soon and very much hope you will all be able to join us and hear about the great work that you are helping to fund.

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