This poster is based on survey data collected from delegates who attended the Fabry Matters Conference in 2024 and offers a platform for RDRP to share insights directly shaped by the patient community.
Initially presented at the WORLD Symposium in February 2025 to an international audience, this research is now being shared with clinicians, researchers and pharmaceutical representatives across the UK.
The key findings of this research were:
Lack of awareness:Â A majority of respondents were unaware of when clinical trials were taking place, mainly due to difficulty accessing information.
Information sources: Those who were aware typically received details from their Fabry specialist and the MPS Society.
Patient needs:Â To improve the patient experience, participants highlighted the need for regular updates, flexible schedules, and emotional or financial support.
Barriers to participation:Â Side effects and travel demands were identified as major barriers to participation.
Recommendations:
To address concerns about side effects or risks, solutions include educational strategies such as providing guides on managing side effects, offering information on pharmacological support, and using expert-led videos to explain safety measures.
To address travel-related concerns, recommendations include locating trial sites closer to patients, and providing comprehensive logistical support, such as assistance with transportation, accommodation, and emotional support to alleviate travel-related stress.
This research highlights the voices of the Fabry community and supports our mission to improve the inclusivity and accessibility of clinical trials.
