See all the latest news, blogs and updates from the medical sector.
News
MHRA consultation on rare disease therapies
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has launched a public consultation on proposed new guidance for rare disease therapies.
27/05/2026
News
MPS Society responds to key government consultations on support for children with complex needs
Over the past few weeks, the MPS Society has submitted responses to two important government consultations focused on education and support for children and young people with complex medical and additional needs.
21/05/2026
Blog
"It's good to know I'm not alone": the value of our community spaces
Our online community spaces continue to grow, with more than 200 members in our WhatsApp communities and over 300 members in our Facebook community. We asked members about their experiences and the feedback was overwhelmingly positive.
21/05/2026
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
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.
