See all the latest news, blogs and updates from the medical sector.
Blog
Derralynn's story - Fabry Awareness Month
This Fabry Awareness Month, we are featuring a range of articles from guest bloggers describing their relationship with Fabry disease....
05/04/2022
Blog
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.
01/04/2022
Story
Steven's story - Fabry Awareness Month
This Fabry Awareness Month, we are featuring a range of articles from guest bloggers describing their relationship with Fabry disease....
31/03/2022
Condition
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.
21/03/2022
Story
Living life with MPS IVA - Joanne's inspirational story
Joanne was diagnosed with MPS IVA Morquio when she was three years old and her mother Judith Evans, one of our lovely trustees opens up...
09/03/2022
Blog
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...
01/03/2022
Story
Why Amy is a unique lady
Amy Cooper, from Hampshire, is 35 years old and has MPS I Hurler. She is one of the few people who underwent a Bone Marrow Transplant at...
28/02/2022
Blog
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...
16/02/2022
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...
