See all the latest news, blogs and updates from the medical sector.
News
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
News
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
News
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
News
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.
24/11/2025
News
Lessons from Taiwan
Sophie Thomas shares the latest updates from the Newborn Screening Summit & 6th APAC Educational Forum for Families and Caregivers on MPS in Taiwan.
20/11/2025
News
Get involved in research more easily with RDRP's research registration
Help shape the future of rare conditions by signing up to take part in research. Find out why it's important and how to hear about research opportunities.
24/10/2025
News
Bob Stevens selected for prestigious 2026 PAL Award
After reviewing numerous nominations and considering many amazing individuals, the WORLDSymposium 2026 Awards Committee has selected Bob Stevens as the recipient of the 2026 Patient Advocate Leader (PAL) Award.
06/10/2025
News
Update from Bob's US tour
This week, our Chief Executive Bob Stevens and RDRP colleague Bennie Marshall-Andrews have been on a vital whirlwind tour of some of our pharma partners in the USA. Bob shares some important updates from the trip.
