Rare Disease Disorders and CNS Drug Development – Paving the Way for Precision Medicine
In CNS drug development, more and more diseases once viewed as common are now known to be collections of rare variants. Our deepening understanding of the human genome and pathophysiology has led to fragmented classifications of common neurological or psychiatric disorders based on identified genetic markers. This trend of replacing larger classifications of common disorders with a more precisely defined spectrum of individual rare and ultra-rare diseases is changing the face of clinical research and development, not only in CNS but across virtually all therapeutic areas. What were once syndromes are now collections of diseases with common expression. Rare diseases are showing the way toward precision medicine for common disorders.
In this webinar, medical experts from Medpace’s neuroscience team explore the following topics:
- The shifting clinical development landscape – Discussion of new genetic findings in Parkinson’s Disease, Dementia, and ALS, as well as the heritability of psychiatric disorders
- The history of treatment of rare diseases with CNS manifestations – successes and failures
- Operational lessons-learned in identifying, enrolling and retaining rare disease populations in clinical trials
- New approaches to deliver disease specific targeted therapies to the CNS