See all the latest news, blogs and updates from the medical sector.
Blog
Emma's story | The Big Give Christmas Challenge 2023
Hello, everyone! I am Emma, mum to my wonderful daughter Isabella. Isabella has MPS I Hurler and we have been members of the MPS Society since 2016.
14/11/2023
Condition
SMC do not recommend pegunigalsidase alfa (Elfabrio®) for adult Fabry patients
SMC do not recommend pegunigalsidase alfa (Elfabrio®) for adult Fabry patients.
13/11/2023
Condition
NICE approves life-changing medication for treatment of alpha mannosidosis
NICE have recommended velmanase alfa as an option for treating alpha mannosidosis, if treatment is started in people under 18 years (it can be continued in people who turn 18 while on treatment).
10/11/2023
Blog
Angela's story | The Big Give Christmas Challenge 2023
Angela's story shines a light on the lasting friendships and strong support networks created through finding a place of acceptance and empathy.
06/11/2023
News
Together we can transform lives
Read our latest Fundraising Round Up and your inspirational stories that enable us to support over 1,750 people every year.
24/10/2023
News
Together we can transform lives
Read the inspirational stories of some of our amazing fundraisers and be introduced to the first of our eight TCS London Marathon runners.
26/09/2023
Story
Believe in yourself
Read Morgan's inspiring story on how becoming an ambassador at her school made her feel more included and proud of herself.
25/09/2023
Story
My daughter Eva
For Leukodystrophy Awareness Month we hear from Catrin, whose daughter has Metachromatic Leukodystrophy (MLD). My daughter Eva Rae...
18/09/2023
Story
My son Joey
For Leukodystrophy Awareness Month we hear from Katie, whose son has Metachromatic Leukodystrophy (MLD). Joey was born a happy healthy...
11/09/2023
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.
