See all the latest news, blogs and updates from the medical sector.
Condition
NICE approves its first ERT for treating Fabry disease
Pegunigalsidase alfa (Elfabrio®) has been recommended as an option by NICE for treating adults with Fabry in England and Wales.
01/09/2023
Blog
Bob's rare disease road trip
Bob has been very busy over the last few weeks, as he travelled across the world to continue the all important conversation on rare diseases
23/08/2023
Blog
Going on holiday in an MPS world
Living with MPS, Fabry or related condition can make going on holiday a daunting prospect. I am here to tell you that it's possible.
01/08/2023
News
Together we can transform lives
Read all about our incredible fundraisers, from marathon runners to mountain climbers and learn how you too can get involved.
27/07/2023
Blog
Thank you Katie and Churchill Park Academy
We would like to say a big thank you to Katie Mackelden who, along with Churchill Park Academy, raised £737.00 during MPS Awareness Week....
04/07/2023
News
Together we can transform lives
Thank you to our amazing fundraisers for the support you have shown us over the past month, let's celebrate your achievements.
03/07/2023
Condition
Gene therapy clinical trial for patients with MPS II Hunter opens in Manchester
A new gene therapy clinical trial for patients with MPS II Hunter disease is opening in Manchester.
16/06/2023
Condition
NICE unfortunately leans towards a ‘no’ for the treatment of early onset LAL D (Wolman disease)
NICE (National Institute of Clinical Excellence), the drug decision making body, have publicly released their interim decision today not...
05/06/2023
Blog
Navigating the NHS by Claire
I may seem a little obsessed, maybe I am, but when you are fighting for your life, for your health, you’ll leave no stone unturned.
29/03/2023
Blog
They had an absolute ball
It was dancing shoes at the ready for the Winter Ball, hosted by the Scott family, on 3rd February, all in honour of their daughter...
