During the recent regional meeting for families and patients living with Duchenne and Becker muscular dystrophy (DMD/BMD), organized by Parent Project in Genoa, Italy on October 26, the Italian organization carried out some engagement activities planned as part of the PaLaDIn project. Specifically, Parent Project involved families in the co-creation of tools for assessing quality of life in Duchenne muscular dystrophy.
The PaLaDin project aims to develop the Interactium®, a global platform for collecting data reported directly by patients with rare neuromuscular diseases. The overall goal is to empower patients’ voices at all stages, from research to treatment planning to the entire drug life cycle, by promoting an integrated approach to patient data. Parent Project participates in PaLaDin thanks to the experience gained with the Duchenne and Becker Patient Registry, active since 2008. Thanks to this wealth of knowledge and the trust built up over more than fifteen years of working with the community, the association brings a valuable contribution to the project, both in terms of collecting reliable data and in its ability to actively involve families.
At the meeting in Genoa, the process of compiling the first questionnaires by patients and caregivers was launched. This is an important step in giving a direct voice to people with DMD and their families, through tools known as PROMs (Patient-Reported Outcome Measures), which collect experiences, difficulties, and the real impact of the disease on daily life.
PROMs are data reported directly by the patient, without interpretation by a doctor or other healthcare professionals. They may relate to the patient’s health condition, their perception of the disease or treatments used, and the impact of these on their quality of life.
The questionnaires introduced in this first phase are specifically designed and validated for the Duchenne population, with the aim of accurately measuring the quality of life of people with DMD and their caregivers.
These data, which are essential for clinicians, researchers, and regulatory and health authorities, provide a better understanding of what really matters to patients and helps guide health policies toward more effective interventions focused on real needs.
Each participant’s contribution will help improve DMD assessment tools, making them more relevant to everyday life and more useful for research and treatment. These early inputs not only feed into the PaLaDIn Interactium® data platform but also demonstrate how patient participation generates measurable value in the co-design of healthcare tools, guiding the upcoming steps of the project and strengthening its patient-centred direction.
