Latest News

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MPS awareness in the news

You’ve been brilliant this MPS Awareness Day spreading the word about these diseases and paying homage to your MPS heroes. As well as a great social media campaign across Twitter, Facebook and Instagram we also reached new audiences through press

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New oral therapy for Fabry patients in development

Idorsia Pharmaceuticals, a Swiss biotech company, is developing lucerastat, a new oral therapy for patients with Fabry Disease. The Phase 3 clinical study MODIFY has just enrolled its first patients and will investigate the use of lucerastat monotherapy for the

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GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics

Agreement strengthens Orchard’s position as a global leader in gene therapy for rare diseases GSK takes 19.9% equity stake in Orchard and seat on board GSK and Orchard Therapeutics have announced a strategic agreement, under which GSK will transfer its

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Ramblings of a CEO

Why do we do what we do? Well there are many reasons that we work in the rare disease space; money, travel, career, passion, belief that we can make a difference. I recently had some training on health economics and

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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