17 | The evolving role of patient organizations in neuromuscular research: integrating the patient voice through registries and collaborative initiatives

Ilaria Zito, F. de Angelis, F. Mattei | Parent Project aps, Roma, Italy.

Over the past decades, the role of patient organizations in neuromuscular research has evolved from passive involvement to strategic partnership. Since its founding in the mid-1990s, Parent Project aps has become a key player in advancing care and research for Duchenne and Becker muscular dystrophy (DMD/BMD), promoting a patient-centered approach to research, aiming to ensure that the priorities of patients and families are fully reflected in clinical and scientific strategies. A cornerstone of this effort is the Italian DMD/BMD patient registry, which has supported clinical trial recruitment, feasibility assessments, and the identification of unmet needs across different regions. Data from the registry have helped shaping advocacy efforts and targeted projects - including Parent Project on the Road (home-based respiratory monitoring), telecardiology consultations initiated during the pandemic, and the launch of a series of international clinical workshops focused on key topics in patient management, particularly relevant for adult patients in the evolving therapeutic landscape. Soon, we will integrate Patient Reported Outcome Measures and Patient Preferences into the registry, offering deeper insight into disease progression, treatment responses, and quality of life. This will further strengthen the registry's role in shaping research priorities and care strategies. This approach underscores the strategic value of involving patient organizations in the design and implementation of research and data collection efforts. It proves especially effective in large-scale, multi-partner research projects, where close collaboration between patient organizations and scientific partners is key to ensuring that research goals remain aligned with real-world patient needs and lead to more effective and equitable outcomes.