Latest News

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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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MPSIV/MPSVI Resilience Workshop held Friday 8th – Saturday 9th March

This workshop was organised and run by the metabolic teams from Birmingham Children Hospital, Royal Manchester Children’s Hospital and Addenbrookes they asked for two advocacy team members from the society to help support the event – Sophie Thomas and Sally

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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