See all the latest news, blogs and updates from the medical sector.
Back to school 2024
Thanks for sharing your new term, back-to-school photos. We love seeing everyone looking so happy!
New beginnings
The summer holidays are always such an exciting time of the year but they can also be the gateway from the familiar to the unknown. Hear from some of our members who have been in the same situation or will be facing new beginnings just like you.
Alison's Scotland trip
The Support and Advocacy Team have been working on building a sense of community across our membership. A huge part of that has focused on Scotland and saw Alison embarking on an epic trip for MPS Awareness Week.
Together we can transform lives
Head over to the fundraising round-up to read about our biggest fundraising achievements to date. You will be amazed at how much has been raised in just 3 months!
SMC accepted pegungalsidase alfa (Elfabrio®) for the long-term treatment of adults with Fabry disease
The Scottish Medicines Consortium (SMC) has accepted pegungalsidase alfa (Elfabrio®), for the long-term treatment of adults with Fabry disease for restricted use.
CSAC Update July 2024
Hear from our trustee, Dr Fiona Stewart, as she reflects on the past six months of CSAC achievements and looks ahead to an exciting autumn.
Update for patients with late onset LAL D (Lysosomal acid lipase deficiency)
Despite best efforts, NICE was unable to make a recommendation for the use of sebelipase alfa in LAL-D patients with non-Wolman disease.
Helen's story | MPS Awareness Week 2024
Helen's preconceived notions of a wellbeing course were shattered once she tried it. The course helped Helen identify positive aspects of her life and recognise the barriers preventing access to that positivity.
Rareminds' counsellors | MPS Awareness Week 2024
The specialist Rareminds counsellors explain the part they play in supporting people living with a rare disease.
Together we can transform lives
We've had some amazing fundraisers taking on a whole array of challenges from head shaves to overseas marathons. It has been great to see so many of you getting your fundraising hats on, read you stories.