G419 – Help Lily Bloom Fund
About This Campaign
Help Lily Bloom was established in honour of Lily, who is living with an ultra-rare genetic condition caused by a mutation in the CASK gene. Fewer than 600 individuals have been diagnosed with CASK-related disorders worldwide.
CASK-related disorders are severe neurological conditions that affect brain development and can impair movement, coordination, communication and other essential functions. There is currently no cure or approved treatment targeting the underlying genetic cause of these disorders.
Two promising gene therapy research programmes are exploring complementary approaches to address the genetic cause of CASK-related disorders. Both are currently at the preclinical stage, where further research is required to establish their safety, optimal dosing and effectiveness before clinical trials can be considered.
