CNS Orphan Diseases - The Alternative Route in CNS 2014

In highly prevalent disorders, such as Parkinson’s or Alzheimer’s disease, there is significant inter-patient variability in symptoms. In the case of orphan CNS indications, the cause of the disease can usually be traced to a single gene. This makes it easier for drug developers to identify targets for those agents, and increases the chance of a disease-modifying therapy being developed.

SMi's masterclass hosted by QCTR will look at the challenges and potential advantages of developing drugs in CNS orphan diseases, compared to larger CNS indications and also looks at the support that is available to drug developers in orphan diseases. It will look at case studies of drug companies who have followed this route.

Key benefits to this masterclass are:
- This masterclass is essential attendance for anyone who wants to understand clinical trials in a rare CNS
disease
- Gain timely insight into key trial design strategies
- Discover the benefits of orphan disease regulations
- Learn from real-life case studies of how companies have approached these challenges