The group of metabolic disorders collectively called mucopolysaccharidoses are rare conditions. This term encompasses conditions such as Hurler syndrome, Hunter syndrome, Hurler–Scheie syndrome, Scheie syndrome, Sanfilippo syndrome and Morquio syndrome, amongst others.

On 25th June 2018 Dr Michelle (k/a Mikhaila) Muscat held a joint interview with Mr Bob Stevens, Group Chief Executive of the Society for Mucopolysaccharide Diseases (MPS) and Ms Sophie Thomas Head of Advocacy & Patient Service.

The MPS society is a registered charity and was founded in 1982 by Christine Lavery and has been operating for 36 years. They support people of all ages affected by MPS and related diseases. Fairly uniquely they also support people with Fabry, another rare disease, hence giving that community a voice as well.

They explained how they have been fundamental in championing patient needs, having an advocacy service which provides critical support ranging from general support as well as gateways, an approach that has been adopted worldwide within the Lysosomal Storage Disease (LSD) communities. They described how they respond to patient needs to make their life easier and how the society also communicates to the general public. The MPS society provides advice and support on housing, social care, education as well as bringing patients together and empowering them.

The society is very active internationally on mucopolysaccharidoses matters. They are part of the international MPS network and MPS Europe. They form part of the LSD collaborative, where all the chief executives come together. Individually the diseases are rare but collectively they become more vocal and may help each other in securing further treatment initiatives.

The MPS society ran several events such as a national conference every 2 years. They had an annual tree planting as well as patient expert meetings. The society maximises the information given to various people. Other activities included host family events, trips to theme parks and Euro Disney, sibling events and young adult weekends. To help make this possible a pool of volunteer carers were enlisted in the societal activities.

The International MPS Symposium was run every 2 years, and they have taken families to some of the international meets together with some volunteers. They ran a young ambassador team with whom they went to places such as Australia and Brazil.

Their subsidiary, MPS Commercial, was involved in the logistics for many trials for rare diseases over the world and operates in every time-zone. They also help the patient with accommodations, re-imbursements and relocations.

They stated they have funded over 6 million pounds worth of research since 1982. This included biomarker research and support with regards to MPS 2 mutation research. They were also helping to fund the clinical trial of Genistein for Sanfilippo Disease through the MPS society patient groups.

Mr Stevens stated that they fully understand the rare diseases they support, especially as the dad of two boys with Hunters he knows the services since he used them and that they are ‘the difference between coping and not coping’. He is very proud of the advocacy services they provide, some of the astounding results they achieved and the dedication and commitment of other members of staff.

 

Further information may be found on their website: www.mpssociety.org.uk