development of a new Patient Needs Questionnaire

In France, two associations actively represent Fabry patients, participate in and promote medical research: Association des Patients de la Maladie de Fabry (APMF, and Vaincre les Maladies Lysosomales (VML, In a patient-centric approach, involving these associations in developing a patient needs instrument is highly recommended to provide a patient perspective.

In this context, the authors identified the need to develop a questionnaire to evaluate and measure treatment expectations for patients living with Fabry disease to heighten the clinical picture and allow physicians tomanage the treatment more astutely.