News from WORLDSymposium 2019

WORLDSymposium 2019 header

News from WORLDSymposium 2019

All the news from the MPS Society attending WORLDSymposium 2019 4-8 February.

WORLDSymposium™ is an annual research conference dedicated to lysosomal diseases. W.O.R.L.D. is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium has grown to an international research conference that attracts over 1600 participants from more than 50 countries around the globe.

8 February – what is the answer?

As WORLDSymposium 2019 draws to a close what are the key messages?

Science has come a long way but it is clear that it still has a long way to go in perfecting new treatment options for rare diseases. Gene therapy is once again proving more complicated to get right than we thought. Many initial reports from trials were somewhat disappointing, but that is going to happen. We all got too excited about gene therapy and thinking it’s the answer and it is here already. It may be one answer but many of the technologies need a lot more work before we can say for sure. There are other answers such as new and existing ERTs. I think they will play a major role in the foreseeable future and we know for many they are a good treatment option. For me one of the most exciting advancements is oral therapies. We know they are a good option for some patients and give them more freedom, but there are psycho social issues to consider as well.

Pharma and science are doing great things and there are bound to be set backs but even these can be considered progress as we learn more about the diseases each time. I understand only too well that time is not our friend in the rare disease community but let us have faith in those working on new treatments. We have some of the best scientific minds working on new treatments for rare diseases and I, for one, am glad for their dedication to finding a way to a better future for the rare disease community.

7 February – MPS Commercial: it’s in our genes

Oli and Sam MPS IIAs I have had meeting after meeting with the pharma industry, especially about new clinical trials, it has become more apparent how important MPS Commercial is to the patient experience of those taking part in these potentially new treatment options and therapies.

Patients who take part in clinical trials are heroes and trailblazers. The science being developed to make these treatments a reality is at the very cutting edge of what is scientifically possible today and I am astounded by how many clever scientists and professionals there are working on these treatments. But, without the patients willing to take part in clinical trials there would be no new potential treatments.

Taking part in a trial can be a daunting thought – not only about the treatment but also how it affects our lives as patients and patient families. MPS Commercial is part of the MPS Society UK so understands rare diseases. We understand what problems families face in taking part in clinical trials because we hear from you everyday. As an MPSII dad I know how stressful living with a rare disease is so with my team one of our key roles is to help patients and pharma understand each other’s needs more clearly.  We undertake bespoke research and survey work to build that important data and patient reported outcomes to enable these new treatments to be approved and reimbursed. This can only be achieved if you truly understand rare diseases and what they entail. That is why we set up MPS Commercial – to help the heroes and trailblazers have more support and understanding whilst being those beacons of light for the future. We take care of the small things such as travel, accommodation, visits to clinical trial centres and of course all the personal expenses that go with taking part in a trial. Yes, these are all small things, but for a rare disease family they are important. To us they are not a number or a profit item on a balance sheet. To MPS Commercial they are our family, our reason to exist and our heroes too. We do this because we care and any profit we make goes directly to MPS Society, for the patient community. Our only shareholders are our patients. This was Christine Lavery’s vision and this is our mission. You may be rare but you are not alone.


6 February – photos!

Jackie at WORLDSymposium with poster

Bob Stevens with FIN

5 February – It’s all about collaboration

WORLDSymposium is not only a great opportunity to connect with our friends and partners around the world but also an opportunity to look at how we can work more closely together with limited resources to improve the lives of the communities we serve. Through MPS Commercial and the MPS Society we are reaching out to support not only our current stakeholders but also to those who do not have a voice yet.

This morning I am taking part in a meeting about Sanofi Genzyme’s Humanitarian Programme. In the UK it can be difficult to get reimbursement for new treatments and therapies. In many countries there is simply no hope of treatments and therapies being made available to those with rare diseases. I, and many others, believe that we have a moral duty to give hope to these communities by working with our pharma partners to provide these treatments and therapies on a humanitarian basis.

The really hard part is that only a small percentage of our rare community  can benefit from these programmes because it is simply not possible to provide free treatment to everyone, but it is a small step in the right direction. These humanitarian programmes can only happen through collaboration and together with my partners from other countries and innovative programmes from industry partners such as Sanofi Genzyme and Takeda (Shire) we are proud to be involved because ultimately it is all about you – rare but not alone.

4 February – WORLD The return!

Well after a long time of inactivity I’m back and so excited to be at WORLDSymposium, Orlando. Whilst I have been away I have not been idle. The MPS Society in the UK has been undertaking an extensive consultation with all our stakeholders about our future. The Vision project, as it has been named, remains true to our founder Christine Lavery’s vision (hence the name) but with a new modern set of objectives. We are so excited as a team but everyone will have to wait until our National MPS Conference on June 28th-30th for the big reveal. In the meantime watch out for updates from WORLD with a bit of fun thrown in. Watch this space!

MPS Commercial poster presentations

We are proud to be presenting four posters at this year’s WORLDSymposium. Click on the images below to see more.

Burden of illness in Sanfilippo disease (MPS III)Pathway to diagnosis in Sanfilippo disease (MPS III)Understanding Fabry in familiesPatient reported outcomes in MPS IVA patients receiving enzyme replacement therapy




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