Rare Disease Research Partners

Book now to benefit from the early bird rate for MPS Matters 2025 this June in Coventry, with rooms starting at £80! This community weekend with expert speakers and interactive workshops for people with MPS and related lysosomal storage conditions and their healthcare professionals, aims to provide useful information for living well with an MPS condition and a chance to meet up with others and share common experiences. Head to our website for more details, the current agenda, and the all-important booking form https://buff.ly/4e3YBCh #MPSMatters2025

As we welcome 2025, we remain dedicated to advancing research, strengthening clinical trial support, and enhancing medical communications. Let us make this a year of meaningful progress and positive change for everyone affected by rare diseases. Wishing everyone a Happy New Year filled with breakthroughs and resilience! #research #clinicaltrialsupport #medicalcommunications #rarediseases #happynewyear

Wishing you all a joyous and festive Christmas from everyone at RDRP! #merrychristmas #research #clinicaltrialsupport #rarediseases #medicalcommunications

The RDRP team will be taking a break during the festive season and our office will be closed. During this time our Clinical Trial Support Team will be available to handle any travel logistics emergencies that cannot wait until our team are back in the New Year. We wish everyone a safe and joyful holiday period. #research #clinicaltrialsupport #rarediseases

Support people and families living with a rare condition and make your Christmas donation count by giving during the Big Give #ChristmasChallenge https://buff.ly/3Vi4qVs Help us reach our new target of £30K and you'll be giving the gift of a community for people who feel isolated and alone because of the rarity of their condition.

We are thrilled to announce that donations to the Big Give Christmas Challenge are officially open ❄️ From now until 12 midday on 10 December your donations made to us via the Big Give website will be matched pound-for-pound; this means your impact goes twice as far! >> https://buff.ly/3Vi4qVs These funds will enable us to organise amazing community events over 2025, so that families affected by rare conditions are able to enjoy fun activities together in a safe environment, accompanied by members of MPS Society staff to provide practical and emotional support. Thank you so much for your support! #ChristmasChallenge

It was an absolute pleasure for Bob Stevens, Group CEO of MPS Society UK, here for those with rare diseases and RDRP, and Bennie Marshall-Andrew, Head of the Clinical Trial Support Team, to catch up with some of the Sangamo Therapeutics, Inc. UK team last week.

Following from last month’s #Spotlightonrare we are now drawing our attention to Mucolipidosis type III (ML III) which is one of the lysosomal storage diseases and is sometimes referred to as Pseudo-Hurler polydystrophy. ML III resembles a less severe form of MPS I Hurler and was first described by Dr Maroteaux and Dr Lamy in 1966. Babies may show little sign of the condition, but symptoms start to appear as more and more cells become damaged by the accumulation of unwanted molecules. You can find more information about the condition on our website https://buff.ly/4eFvaWI #Spotlightonrare

We’re delighted to announce that RDRP's abstract on the barriers and facilitators to clinical trial participation has been ACCEPTED for poster presentation at #WORLDSymposia 2025. Well done to all involved in this fantastic research and we can’t wait to see the full poster presentation at #WORLDSymposia #Research #FabryDisease #ClinicalTrials

Our online resources are there for you to find helpful information and further insight into the conditions we can support you with https://buff.ly/3YTqOqy Today we are revisiting a research project published by MPS Society and Rare Disease Research Partners authors on the educational journey of individuals with MPS IVA Morquio.