See all the latest news, blogs and updates from the medical sector.
Condition
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...
04/02/2022
Condition
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...
02/02/2022
Blog
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...
31/01/2022
Condition
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...
07/01/2022
Story
January Fundraiser of the Month - Marina and Friends
We were so sorry to hear that Marina passed away in January 2023, our thoughts go out to her family. Marina accomplished so much for the...
04/01/2022
News
Think Rare, Think MPS
The Society for Mucopolysaccharide Diseases (MPS Society) is committed to bringing about change in the diagnostic journey of children...
17/12/2021
Blog
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.
23/11/2021
Story
Lucy's story - The Big Give Christmas Challenge 2021
Lucy tells us why support services from the MPS Society are so important and how you can double your donation to fund them this Christmas.
23/11/2021
Story
Dan's story - The Big Give Christmas Challenge 2021
Dan tells us why support services from the MPS Society are so important and how you can double your donation to fund them this Christmas.
15/11/2021
Condition
NICE leans towards a no for the treatment of MPS IVA (Morquio A)
NICE (National Institute of Clinical Excellence), the drug decision making body in England, have publicly released their interim decision to
