As per the doctors, Charlotte was suffering from Batten Disease. The medics had also ... suffered from the conditions as well, however, its symptoms didn't start to show up when they were given ...

Neurogene reports interim Phase 1/2 trial results for NGN-401 gene therapy in treating pediatric Rett syndrome, showing ...

A serious adverse event in the New York biotech’s phase 1/2 trial of its Rett syndrome gene therapy puts a question mark over ...