Latest News

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Ramblings of a CEO

Well it has been just over a week since the 2019 MPS National Conference and as I sit here whilst on leave in Mallorca with my family I have had the chance to reflect on the weekend. I wanted the

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Orchard Therapeutics have shared updates on their disease programs and first quarter financial results

Orchard Therapeutics, a leading pharmaceutical company has announced progress made advancing gene therapy aimed at correcting neurometabolic diseases and has also provided reports on their first quarter 2019 financial results. Earlier timeline for two disease programs; Clinical Trial in Sanfilippo

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Participants needed for medical market research on Fabry Disease

We are looking for people who are over 18 and have been diagnosed with Fabry Disease to participate in a study we are currently conducting. The purpose of the research is to understand your experience of living with Fabry. Full

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Interview with Michelle Wood

Michelle Wood is the Metabolic Physiotherapist at Great Ormond Street Hospital, we asked Michelle if she would answer a few questions about her work with us and our members.   In a sentence what is your job role? I am

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MPS in numbers

896

individual and families
supported in 2015

25

rare disease supported

3,364,628

£ given research projects
between 1984-2010