International Niemann–Pick Disease Alliance

NP-UK’s 22nd Annual Family Conference – until next time!

NP-UK, a member of the INPDA, held their 22nd Annual Family conference this weekend in Bedfordshire, UK. Dr Will Evans, dad to Sam (NP-C) and a trustee of NP-UK gives this summary:

This year’s conference has come to a close. It was another great opportunity for those involved with these diseases to meet, support one another and share new knowledge and experience.

On Friday during the 6th Interactive Workshop, professionals, clinicians, basic scientists and pharmaceutical company representatives, came together in an open and relaxed atmosphere to discuss the latest developments. We heard exciting reports of new research, from both experienced & new to the field researchers, from the Universities of Oxford, Cardiff, Aston & Heidelberg in Germany.

With upcoming trials in both NP-C, (Orphazyme’s Arimoclomol and Vtesse’s VTS-270), and ASMD NP disease, (Genzyme’s Olipudase alpha), it was a timely opportunity to discuss what is happening and why. With a further presentation from CTD Holdings of their early ideas for a clinical trial of Trappsol, there was lively debate on the relative potential merits of each approach.

On Friday evening, delegates for our 22nd Family conference arrived.  With old friends and new from across the UK, Norway, Belgium, Canada, Brazil, Australia and the USA, it was a great opportunity to catch up and share experiences. Over the weekend there were sessions covering basic science, clinical trials and care for those affected by these diseases.

The children and young adults had their own activity programme, with an outing to Wicksteed Park, and on Saturday evening the “Roaming Reptiles” visited, complete with two very popular meerkats.  

With much talk of the upcoming trials, it is an exciting but challenging time for families. We hope that the weekend enabled families to get as much information as possible to feel empowered and supported in meeting these challenges.

As always, if you need more information, or just want to talk things through, you can contact us via the NP-UK office on (+44) 0191 415 0693, email info@niemann-pick.org.uk, or find us on Facebook Niemann-Pick UK

We look forward to the year ahead with great excitement.

Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

Think Again. Think NP-C

Join our international campaign to improve diagnosis of NP-C!