An update for patient associations and families from BioMarin April 2017 BioMarin have received a number of enquiries regarding the status of their clinical development program in Sanfilippo Syndrome Type B (MPS IIIB). The program, which involves multiple centres around
To celebrate MPS Awareness Day 2017 we are offering lucky families the opportunity to visit Gulliver’s Theme Park in Milton Keynes on Sunday 14th May 2017. If you’re looking for a family fun-filled day out for you and your little ones
This Fabry Awareness Month, you can join Rare2Aware live on Twitter to explore the impact that Fabry disease has on patients and their families, and to discuss the importance of talking to your relatives about your Fabry family tree. When?
Due to the lack of response to our initial press release, which was released three hours before the tragedy in Westminster on 22 March 2017 and understandably was not a priority at this time, we are reissuing this press release.
individual and families
supported in 2015
rare disease supported
£ given research projects