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 aimed 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 hoped to 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, nizubaglustat, in this patient group.
PRONTO was designed as a multinational study and took place in 6 countries, with 31 participants between the age of 2 to 20 years with neurological disease onset after the 1st birthday. Enrolment was initiated in 2021, completed this year, and all participants will remain within the follow-up period until 2025.
If you are interested in learning more about the PRONTO, you can access the explanatory video and further details on the natural history trial here: Azafaros for Patients/Patient Resources and Information.