Symptomatic vs Genetic Disease Modifying Treatments

Time: 9:10 am
day: Day One


Most industry portfolios reflect either a strategic balance between, or unitary commitment to either small molecule or genetic modalities for engaging targets involved in the biological processes implicated in rare diseases of the central nervous system. This panel will explore key strategic, technical, and scientific considerations shaping these decisions.

• How can we assess benefit/risk potentials of therapeutic modalities, and tailor them for patient subtypes?

• How has our increased understanding of genetic mechanisms sharpened the field’s rationalization of drug targets and improved opportunities for disease modifying approaches?