See all the latest news, blogs and updates from the medical sector.
April Fundraiser of the Month - Steven Gill
Steven Gill shares his connection to Fabry and why he's chosen to take on a huge fundraising challenge.
NICE approves life-changing medication for treating MPS IVA Morquio
Elosulfase alfa (Vimizim®) has been recommended by NICE for treating MPS IVA Morquio in people of all ages in England and Wales.
March Fundraiser of the Month - The Hampden Arms
For our 40th anniversary this year, we have decided to have a 'Fundraiser of the Month' throughout the year. This month, we have The...
Why Duncan is running a marathon a month in 2022
Discovering that a relative has been diagnosed with a rare disease can be a bit overwhelming and leave you feeling somewhat worried and...
NICE final guidance approves life-changing gene therapy for treating MLD
We are delighted to announce that Atidarsagene autotemcel (Libmeldy®) has been recommended as an option by NICE (The National Institute...
NICE update on the re-evaluation for the treatment of MPS IVA (Morquio A)
NICE (The National Institute of Clinical Excellence), the drug decision making body in England, have publicly released an update today (2...
February Fundraiser of the Month - Towersey Morris Men and the Haddenham Mummers
For our 40th anniversary this year, we have decided to have a 'Fundraiser of the Month'. This month, we have The Towersey Morris men and...
AVROBIO ends its gene therapy trial for Fabry disease
We are sad to announce that the pharmaceutical company AVROBIO has made the difficult decision to deprioritize its gene therapy trial for...
Think Rare, Think MPS
The Society for Mucopolysaccharide Diseases (MPS Society) is committed to bringing about change in the diagnostic journey of children...
Hannah's story - The Big Give Christmas Challenge 2021
Hannah tells us why support services from the MPS Society are so important and how you can double your donation to fund them this Christmas.