See all the latest news, blogs and updates from the medical sector.
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
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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.
18/03/2026
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Proposed EHCPs reforms and SEND consultation
We want to reassure families that there are no immediate changes to EHCPs and there is no action you need to take at this stage.
03/03/2026
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Bob Stevens receives prestigious PAL Award
At the 2026 WORLDSymposium, Group CEO Bob Stevens was honoured with the PAL Award. In his speech, he emphasized the importance of standing together as a global community and that this work must always be about people not profit or politics.
05/02/2026
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FDA places temporary clinical hold on two MPS gene therapy trials
The MPS Society is aware of an announcement from REGENXBIO regarding its investigational gene therapy programs RGX-111 for MPS I Hurler syndrome) and RGX-121 for MPS II (Hunter syndrome).
29/01/2026
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Raising awareness for childhood dementia event
We need to act now. Read a summary of the key messages from the Childhood Dementia Scotland event in December 2025.
09/01/2026
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UK NSC does not recommend screening for MLD
We are deeply disappointed by the UK NSC’s decision not to add MLD to the newborn screening panel and we will not stop fighting for newborn screening for MLD.
08/01/2026
News
Little Bus Big Ride
To celebrate their family business turning 100 years old, three brothers from Nantwich, Cheshire decided to embark on the adventure of a lifetime.
01/12/2025
News
New hope for children with devastating rare genetic disorder, thanks to world-first research in Manchester
The parents of a three-year-old boy born with a devastating, life-limiting genetic condition say they are now excited for his future after he received a revolutionary stem cell gene therapy treatment.
