What a busy month May has been! The MPS Society team continues to champion your voice across a range of national conversations, collaborations and awareness initiatives. Here's a summary of how we've been representing you throughout May.
Building a research and innovation ecosystem for rare diseases
This webinar highlighted the urgent need to better integrate European Reference Networks (ERNs) into national health systems to accelerate access to orphan medicines and rare disease treatments. Key themes included:
Leveraging health data spaces
AI
Collaborative frameworks (like the European Partnership Programme) to create a unified, innovation-driven ecosystem for rare disease research across Europe
Voluntary and Community Sector Forum meeting
Key Priorities for the next six months were discussed at the most recent Voluntary and Community Sector Forum meeting. NICE aims to improve uptake and adoption of guidance and strengthen their reputation and influence. We are looking forward to see their increased use of patient story telling, use of animations and simplified language to make their content more accessible.
NICE & carers: Public involvement workshop
Sophie took part in a valuable workshop with NICE focused on carers and public involvement. Elevating the voices of carers is crucial, as they play an essential role in navigating complex care systems. Through this workshop, we’re helping NICE shape policies that are more inclusive and informed by lived experience.
ABPI workshop
Bob participated in an important workshop hosted by the Association of the British Pharmaceutical Industry (ABPI). The session focused on accelerating access to treatments and fostering stronger industry-patient collaborations. Representing your interests at this level ensures that patient needs remain a top priority in conversations about future healthcare innovations.
Expert consensus on digital technologies
Bob also contributed to an expert consensus initiative exploring the role of digital technologies in rare disease care. As digital tools become increasingly integrated into healthcare, it’s essential that their development reflects the real-world experiences of patients and families. This work will inform future guidance and policy on digital health within rare diseases.
MPS Awareness Week networking event
One of the highlights this month was MPS Awareness Week, culminating in a vibrant MPS Awareness Day networking event in London on 15 May. This event brought together professionals, researchers and families to shine a light on MPS and related conditions. The energy and commitment from everyone in the room reinforced the power of community, collaboration and continued advocacy.
Looking ahead
Whether it's through workshops, collaborative research initiatives or awareness events, representing your voice remains our top priority. Every opportunity to connect, influence and inform helps drive the change needed for individuals and families affected by MPS, Fabry and related rare diseases.
