Patient Partnership

Patient initiative is the driving force behind ProDGNE, with the aim to fully incorporate patient partnerships and perspectives and ensure patients have a primary role in guiding and identifying research priorities. ProDGNE was developed considering the patient journey, symptoms, concerns, the impact of the disease and what matters to all patients at different stages of the disease. The input and voice of patients is central to the project, with full synergy between all partners being essential to ensure questions and results are relevant in all aspects of research. Patients’ perspective will be of critical importance in defining drug features and characterising the desired therapy and trial design. 

ProDGNE aims to ensure early dialogues between patients and researchers, and co-create an innovative therapy for GNE Myopathy where patients are central partners in research. Patients share common expectations for future therapies and report that they do not wish to lose other functions or abilities. ProDGNE will address these needs by developing an easy to administer oral treatment to stop or slow disease progression and limit the impact of disease on quality of life and autonomy.