SOURCE: Xtalks

Xtalks Webinars

October 24, 2017 07:30 ET

Rare Disease Disorders and CNS Drug Development - Paving the Way for Precision Medicine, New Webinar Hosted by Xtalks

TORONTO, ON--(Marketwired - October 24, 2017) - On Wednesday, November 15, 2017 join medical experts from Medpace's neuroscience team as they explore:

  • 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

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.

Family history has long been central to clinical assessment of neurological and psychiatric diseases. Genetics has moved from disease risk factor to potential etiology in small patient subgroups, demanding a tailored approach to drug development. In neurodegenerative diseases, this shift is well underway. In psychiatry, responsible genes have been found in fewer syndromes, but the evidence for heritability and new ways of dividing populations by symptom complex, rather than classic diagnosis, are gaining ground and pointing the way to new approaches.

This improved understanding of the genetic basis and biochemical pathology of disease in genetic variants has combined with a revolution in bioengineering to potentially deliver innovative products directly to the CNS, thereby addressing unmet needs in neurological and psychiatric diseases with highly targeted therapy. This rare disease approach is beginning to impact patient care, and provide important opportunities for future clinical development in neuroscience and psychiatry.

For more information about this complimentary event, visit: Rare Disease Disorders and CNS Drug Development - Paving the Way for Precision Medicine.


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