Advanced filters
Your story
I’ve always loved sharing stories that move people. That’s why I’m so excited to be releasing my…
Your story
Back in 2018, when I met up with MDUK to talk about the charity and its origins, I was absolutely…
Blog
Good luck Gitte
Three years ago, keen runner Gitte was diagnosed with myositis, a rare auto-immune…
News
We reported last year that Fulcrum Therapeutics announced that it will be starting a phase 3…
Your story
Josie is a fun loving, artistic five-year-old who loves school, swimming, playing with her little…
Blog
Emery-Dreifuss muscular dystrophy (EDMD) affects approximately one in every 250,000 people. EDMD is…
Your story
I’ve always been very active – I think that’s why being in the Fire Brigade suited me so well.
I…
Your story
When I was diagnosed with myositis – a rare auto-immune disorder that involves chronic inflammation…
News
On January 9th 2023, following a small number of children having suspected significant adverse drug…
News
Zolgensma (Onasemnogene abeparvovec) is now recommended as an option for treating presymptomatic…