Caregiver Perspectives Wanted on Future Genetic Therapies for Fragile X

We are keen to hear from you what your thoughts are on gene therapy and its potential use in Fragile X Syndrome (FXS).

While various interventions to help people with FXS are available, there are currently no medicines or other therapies that target the underlying genetic change which causes the condition.  Gene therapy represents a different therapeutic approach as it specifically targets the underlying genetic factors which lead to FXS.

Early studies in laboratories have shown that gene therapy might be helpful in FXS, though at present no research has been done in people. However, there are human studies of gene therapy being done in other genetic neurodevelopmental conditions (e.g. Rett syndrome) raising the possibility that gene therapy for FXS might become a reality in the future.

We are aware that for some people this could be a controversial subject, so we think it is important that families affected by FXS get the chance to give their views about gene therapy.  We are therefore grateful to you for answering the following questionnaire.


You can find out more about gene therapy in general here