Study to Understand Fatal Genetic Diseases in Children Incorporates Digital Participant Education Program
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A clinical trial was launched last week to help researchers understand how two rare and fatal genetic diseases develop in children and provide insight into treatment for them in the future. The study, run by Azafaros, will follow patients in the UK, US, France, Italy, Germany and Brazil for four years.
The PROspective Neurological Disease TrajectOry study (PRONTO) is a Natural History Study that follows children who have been diagnosed with GM1 or GM2 gangliosidoses (also known as Tay-Sachs and Sandhoff disease), a class of rare inherited disorders that are fatal in childhood and which currently have no approved treatments.
