The aim of this project is to gather the stories and lived experiences of patients and caregivers within the Morquio B and GM1 gangliosidosis community to create a core list of medical and quality-of-life outcomes. Our team wishes to empower individuals from the community to take part in research that is meaningful to them. It is just as important and necessary for researchers to learn information about what is impactful to people with Morquio B as it is to research medical symptoms and issues.
Developing a list of outcomes will allow future clinical trials to align better with the interests of the Morquio B community and health professionals. This is a continuation of the previous research projects on the natural history of Morquio B and GM1 supported by the Priest Family Fund for Morquio B.
Currently, we are inviting members from the international community to join our focus group sessions to discuss their experiences with us, and to share what they consider to be helpful treatment end goals.