3rd Rare CNS and Neurodevelopmental Drug Development Summit

The 3rd Rare CNS and Neurodevelopmental Drug Development Summit is dedicated to symptomatic and disease modifying treatments across neurodevelopmental disorders.

Topics

• Specific therapeutic modalities including small molecules, gene therapy, antisense oligonucleotides, gene replacement, stem cell therapy and hybrid strategies
• How to leverage precision medicine to treat rare neurodevelopmental diseases
• The development of translational and quantitative biomarkers to strengthen sensitivity and specificity of outcomes measures
• Symptomatic and disease modifying treatments and how our increased understanding of genetic mechanisms has sharpened our thoughts on targets and disease modifying approaches