Completed

PRONTO Study

PRONTO Natural History Study

PRONTO (PROspective Neurological Disease TrajectOry Study) was a natural history study designed to better understand disease progression in children with late- infantile and juvenile-onset GM1 and GM2 gangliosidosis. The study focused on neurological signs and symptoms and aimed to identify patient characteristics that may help explain and predict disease progression.

The completed study has generated an external control dataset and informed the development of a disease progression model, supporting future clinical trials of nizubaglustat in this population.

For more information about PRONTO, you can access the explanatory video.

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