Latest Fragile X Clinical Trial and Research Updates | May 2024 Webinar

[@wordswords2094]

Please post ways to find Direct to Consumer testing. It is almost impossible for many to get to a PCP and then to a geneticist, and then to get the testing. There is a level of privileged inherent in that path which many don't possess.

[@andreakonickova6700]

Hi is there any chance the drug could be used for fragile-x premutation?

[@FRAXA]

Thank you for reaching out with your question. The investigational new drugs we're discussing are specifically being tested for treating Fragile X syndrome. While they offer hope in this area, they are not currently anticipated to be particularly effective for individuals with the Fragile X premutation.

[@scentedgamer8676]

How long do the trials last?

[@FRAXA]

Phase 3 clinical trials typically last between one to three years. This phase involves a larger group of participants and focuses on confirming the effectiveness of the treatment, monitoring side effects, and comparing it to standard or placebo treatments. The exact duration can vary based on the specific objectives, the number of participants, and the endpoints being studied.