See all the latest news, blogs and updates from the medical sector.
Blog
Sally's story | The Big Give Christmas Challenge 2023
Meet Sally, one of our Senior Advocacy & Support Officers. She has facilitated and joined many community events and treasures the time spent together and being able to connect face to face with our members.
21/11/2023
Blog
Today is World Sanfilippo Awareness Day
MPS III, known as Sanfilippo disease, is one of the mucopolysaccharide storage diseases.From 2010 to 2020, 83 babies were born with MPS III in the UK.
16/11/2023
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
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
