PRAX-100 / Praxis Precision Medicines, The Florey Institute of Neuroscience and Mental Health - LARVOL DELTA

Additional Pipeline Updates (GlobeNewswire) - "Praxis remains on track to nominate a development candidate for each of its early stage ASO therapeutic initiatives in 2025: PRAX-080: Focused on targeting PCDH19 mosaic expression which represents a pioneering approach to treating PCDH19-related epilepsy, a rare but devastating genetic disorder characterized by early-onset seizures and cognitive impairment, disproportionately affecting females. PRAX-090: Designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs. PRAX-100: Targeting SCN2A LoF mutations, the predominant genetic link to de novo autism spectrum disorders (ASD)."

Pipeline update • CNS Disorders • Epilepsy