Latest News

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The evaluation of Velmanase alfa for treating alpha-mannosidosis has been put on hold.

This HST evaluation has been put on hold as of 2 October 2018. The company which markets this technology (Chiesi) are currently developing an offering for submission to NHS England. Once confirmation of the submission has been received NICE will

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Trip advise us

We always want to hear from you on the best places you have found for holidays and trips away, whether they have given you the best support and guidance or just made a few suggestions along the way making the

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Update on genistein trial for patients with MPSIII Sanfilippo

High dose genistein treatment in children with Sanfilippo disease shows no clinically meaningful benefit A Phase III, Double Blinded, Randomised, Placebo Controlled Clinical Trial of High Dose Oral Genistein Aglycone in Patients with Sanfilippo Syndrome (Mucopolysaccharidosis III A, B and

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New beginnings for the international MPS network

Meeting in San Diego at the 15th International Symposium on MPS and related diseases, over 20 of the 40 patient organisations that form the International MPS Network (IMPSN) came together to breathe new life into the group. The aim of

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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