The purpose of the study is to collect opinions about gene therapy research from people diagnosed with a genetic ataxia from a number of different countries. People with genetic ataxias can complete the survey, or family members can complete it on their behalf, with their consent.

Genetic forms of ataxia are caused by mutations in one or more genes. The mutated gene responsible for the ataxia is passed along from generation to generation by family members who carry it. Some of the common forms of genetic ataxias are Friedreich’s ataxia (FA), ARSACS, Spinocerebellar ataxias type 1, 2, 3, 27B, CANVAS or Ataxia Telangiectasia (AT), and there are many others. Gene therapy is a potential treatment for genetic ataxias. We are interested in learning about opinions to gene therapy from people with all types of hereditary ataxias, as long as the genetic diagnosis has been confirmed. To be eligible to participate, you or your family member must have a clear diagnosis of ataxia caused by a mutation in a specific gene. The results will be used to provide information to researchers developing future treatment trials for genetic ataxias, and to improve patient interactions during the development of such treatments.

This project is being conducted as part of the TREAT-ARCA research project funded by European Joint Programme for Rare Diseases. This project also received funding from the biopharmaceutical company UCB.

Link to Survey in English: https://www.surveymonkey.com/r/CHZZWLD

Lien vers le sondage en français: https://fr.surveymonkey.com/r/NCQ8HXV 

Link al sondaggio in italiano: https://it.surveymonkey.com/r/8SPL6NH 

Link zur Umfrage auf Deutsch: https://de.surveymonkey.com/r/K3TNHVZ 

Disclaimer: This research opportunity is not affiliated with NAF. We are sharing this information as a resource for the Ataxia community. Sharing this information does not imply endorsement by NAF.