Our goal is to develop a guideline that will capture the lived experiences of max 140 patients across multiple LGMD subtypes in order to identify patterns in access to care, treatment satisfaction, and daily quality of life. With this survey the patients’ voice will illustrate the differences between countries, and the results of this survey will be incorporated in an article and will be presented at the November 2026 ENMC workshop on cardiac care. In this way, the survey results will guide future patient engagement strategies and improve clinical communication. It reflect the patient voice in the ENMC workshop and with further implementation of outcome a masterclass for the cardiologists that treat LGMD patients (in partnership with Treat NMD).
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We will anonymize all data before we present and store it at Treat NMD.