See all the latest news, blogs and updates from the medical sector.
Condition
Chiesi receives MHRA approval for additional dosing regimen of Elfabrio
We are pleased to share that Chiesi has received MHRA approval for an additional dosing regimen for pegunigalsidase alfa in adults stable with an enzyme replacement therapy (ERT).
14/05/2026
Story
Ayla's story | MPS Awareness Week 2026
This MPS Awareness Week, we hear Ayla’s story. Diagnosed with MPS I Hurler at the age of four, Ayla is at the heart of a close‑knit and outgoing family unit.
13/05/2026
Story
From hospital ward to centre stage: Sam’s Story inspires new play
We caught up with Sam in March and watched him in a play written by his mum and inspired by his time on a children’s ward.
24/04/2026
Blog
Equity and quality in rare conditions
Improving equity and quality of care for people living with rare conditions continues to be a key priority across health and care systems. It is encouraging to see organisations such as NICE developing clear quality standards for rare diseases.
23/04/2026
News
Benefits support and upcoming changes
We know that ongoing changes to the benefits system can feel unsettling and that many families are facing uncertainty as reforms are discussed and introduced over the coming years.
23/04/2026
Blog
Together we can transform lives
Blue is the colour of our steely steppers all racing for rare. Head over to our fabulous fundraising blog to find out more!
23/04/2026
Condition
Progress in MPS II and MPS III therapies
Clinical research in MPS continues to move at pace internationally, with a number of important regulatory developments over the past few months, particularly in MPS II Hunter and MPS III Sanfilippo.
23/04/2026
News
Denali Therapeutics announces U.S. FDA approval of MPS II treatment
Following Denali's announcement that the FDA has approved an enzyme replacement therapy for the neurological aspects of MPS II, we recognise that our community may have questions.
27/03/2026
News
Leni’s story strengthens call for childhood dementia recognition and newborn screening reform
Leni's story highlights the devastating reality faced by families affected by childhood dementia, why this needs to be a recognised condition and how newborn screening reforms can help others living with rare conditions.
24/03/2026
News
Family living with MPS III join Jesy Nelson for emotional Mother's Day campaign
Emily and Leni were invited to join Jesy for the special Mother's Day event she organised to celebrate mums caring for children with disabilities and rare conditions.
