The SMC decision regarding Migalastat (Galafold) was announced on 07 November 2016. The relevant Detailed Advice Document can be found here.
WORLDSymposium™ announces Christine Lavery, MBE, Group Chief Executive for the Society for Mucopolysaccharide Diseases, as the recipient for the 2017 Patient Advocate Leader (PAL) Award. Please go to www.WORLDSymposia.org for the full announcement.
The MPS Society is pleased to announce that NICE has given an interim recommendation for Migalastat to be used as a treatment option for Fabry disease in patients over the age of 16 years. This is not the final recommendation
Lapland UK is a unique, award winning theatrical event that recreates Father Christmas’ arctic homeland for one month only each winter. As guests of Father Christmas you will be given special permission to enter the secret world of the elves. Help
individual and families
supported in 2015
rare disease supported
£ given research projects