A novel gene therapy, NGN-401, has been administered to treat Rett syndrome in two paediatric patients at Texas Children's Hospital.
Rett syndrome is a rare but severe childhood neurological disorder, which mainly affects girls.
'We remain on track to report preliminary clinical data from the first cohort of patients in this trial in the fourth quarter of 2024, with additional data from an expanded number of patients expected in the second half of 2025,' Dr Rachel McMinn, founder and CEO of Neurogene, the treatment's manufacturer, said.
Read Rett Syndrome News for more detail.
