Rett syndrome: Family Perspectives 2024

We recently made this short but powerful film to support the Taysha Gene Therapies MHRA ILAP application which was approved earlier this year.
The MHRA is our UK medicines and health regulator similar to the US FDA. The Innovative Licensing and Access Pathway (ILAP) aims to make it quicker for companies to bring potential treatments to market, making it quicker and easier for patients to access new medicines.
We would like to give our heartfelt thanks to Claire, Megan and Jayne who were so open and honest in describing how a Rett syndrome diagnoses shatters the whole family and to Taysha Gene Therapies for inviting our contribution and for all their ongoing partnership as we move towards UK clinical trials.