About the PRONTO study

What is PRONTO?
What is involved?
About the PRONTO study

What is the PRONTO study?

PRONTO is a natural history study aiming to understand how GM1 and GM2 gangliosidoses progress over time, which might help researchers understand how the condition develops and possibly how to treat it in the future. In the short video below, you can learn more about what PRONTO is, and why it is important.

Who can take part in PRONTO?

The PRONTO study is looking for children and young adults between 2 and 20 years of age with GM1 or GM2 gangliosidosis to take part.

If the following criteria also apply to you, or your child, you may be able to join the study:

  • Developed neurological symptoms on or after 1 year of age

  • Have problems with walking and balance, and/or have difficulties with speech

  • Willing to complete the study assessments

Unfortunately, you will be unable to take part if you are receiving or due to receive gene therapy, stem cell transplantation, or experimental drugs. Your physician can give you more advice.

If you would like any further information relating to the PRONTO study, please contact the study team by email - pronto@azafaros.com

Provided by

Created in collaboration with families, caregivers and patient advocacy groups.

Date of preparation: May 2022