PRONTO study

Our Natural History study

Prospective longitudinal study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 gangliosidoses, PRONTO. To gain more insight in the natural development of the disease, we designed PRONTO (PROspective Neurological Disease TrajectOry Study), a prospective Natural History study. By enrolling children with late infantile and juvenile onset of GM1 and GM2 gangliosidosis, we aim to understand how the disease progresses, with a focus on how it affects neurological signs and symptoms, and to identify if some patients have different traits that could explain and predict how their disease will progress. In this way we build an external control dataset and develop a model of disease progression. This study will support the future development of a clinical trial investigating the potential of our lead therapeutic candidate, AZ-3102, in this patient group.

PRONTO is designed as a multinational study in up to 6 countries, with at least 75 participants between the age of 2 to 20 years with neurological disease onset after the 1st birthday. Enrollment initiated in 2021, with a follow-up period until 2025.

Join the PRONTO study

By joining this study, you can contribute to collect important data that will be highly valuable for the development of future treatments. If you want to learn more about PRONTO and how to enroll, please contact you treating physician or click here for the clinical trial information.

If you are interested in learning more about the PRONTO study and what participation in the study entails, you can access the explanatory video and further details on the natural history trial here: Patient Informational.