Antisense oligonucleotide (ASO) therapies have recently shown success in neurological diseases. Several companies are now developing ASO therapies for Phelan-McDermid syndrome. Learn more about how ASO therapies work, current programs, and the PMS ASO therapy landscape.

Get an inside look at the lessons, insights, and opportunities from Ralf Schmid’s visit, including more information about an upcoming partner conference with CureSHANK and Asia-Pacific organizations dedicated entirely to PMS.

Current biomarker assessment tools for neurodevelopmental diseases like PMS can be imprecise and subject to bias, making it harder to objectively measure a treatment's impact. CureSHANK is actively working with expects to evaluate promising biomarker approaches that will provide more accurate and objective measurements for PMS research.