We are launching a new study for individuals aged 12 and older with Tay-Sachs or Sandhoff disease in the juvenile or adult-onset form. The PRADO study focuses on the psychiatric and neurological symptoms associated with these rare gangliosidoses.

A particular emphasis is placed on the cerebellum, which plays a key role in movement, speech, perception, and planning. The aim is to identify early changes, evaluate previous treatment experiences, and develop new preventive strategies.

Study procedure:
Participants attend three sessions involving interviews and questionnaires.

With this study, we aim to:

• improve understanding of the disease mechanisms,
• identify early warning signs,
• assess which treatments have proven effective – and which have not,
• develop new treatment strategies for the future.

The PRADO study is an important step toward improving care and quality of life for people living with Tay-Sachs or Sandhoff disease.