See all the latest news, blogs and updates from the medical sector.
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.
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.
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.
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.
My daughter Eva
For Leukodystrophy Awareness Month we hear from Catrin, whose daughter has Metachromatic Leukodystrophy (MLD). My daughter Eva Rae...
My son Joey
For Leukodystrophy Awareness Month we hear from Katie, whose son has Metachromatic Leukodystrophy (MLD). Joey was born a happy healthy...
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.
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
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.
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.