As multiple therapeutics make their way through clinical trials it’s important that all stakeholders involved in the drug development effort hear directly from the Rett syndrome community. Those of us who love and care for someone with Rett syndrome understand deeply what a treatment that lessens or reverses symptoms would mean, and it’s important that we communicate our urgency and hopes to the US Food and Drug Administration (FDA) – the ultimate regulator of new drugs.
That opportunity is coming up on March 11, 2022, at the Externally Led Patient-Focused Drug Development Meeting with representatives from the FDA and other stakeholders, including drug developers.
Parents, family members, and caregivers are experts in Rett syndrome, and this means we have perspectives that are crucial to understanding treatment benefits, risks, and burdens.
During the meeting we will hear from parents of children of different abilities and ages who will share poignant remarks on the challenges and rewards of raising a child with Rett syndrome.
Everyone who participates will have ample opportunity to provide valuable input through polling questions, live comments and questions, or submitting a comment online.
In fact you don’t have to wait until March 11. Submit a comment or question now by visiting this website: https://rettpfdd.org/
The meeting will be virtual, and everyone in the Rett community is invited to participate. The meeting will be recorded and available online for those who can’t join live. To learn more, see the program, and to register go here.