In particular, its leading candidate is hands down TSHA-102 for Rett syndrome. The way this therapy works is by using “self-complementary” adeno viruses [scAAV] to deliver its treatment.

Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...

“The Rett syndrome community in Europe is excited by the prospect of having an available treatment option. Rett syndrome has a significant impact on people living with this condition, and also ...

Rett syndrome is a neurodevelopmental disorder that primarily affects girls, most of whom have mutations in the transcription regulatory gene MECP2. However, mutations in MECP2 also have been ...

Neuren Pharmaceuticals (ASX: NEU) has announced that the US Food and Drug Administration (FDA) has granted Fast Track ...