Amarantus Bioscience Holdings Inc (AMBS)

[blackhawks]

Is PD an orphan indication, I think not. While it is truly not the most common neurologic disease, it is also not uncommon. TBI would not be an orphan indication either.

Sounds like subsets or precursors of the non-orphan diseases can serve as substitute/alternative routes toward proving drug effectiveness.

http://www.smi-online.co.uk/pharmaceuticals/uk/masterclass/cns-orphan-diseases-the-alternative-route-in-cns

The challenges of drug development in CNS is well known, the recent failures of the large phase III trials in Alzheimer's disease illustrates the problem.

There is however an alternative route in CNS which can prove to be more accesible to small and medium sized companies, and that is CNS Orphan diseases. Diseases such as Progressive supraneuclear Palsy (PSP), Fronto Temporal Dementia (FTD) or Huntington's disease can be initial substitutes for the likes of Alzheimer's disease or Parkinson's disease in which to test the effectiveness of a drug.

This masterclass 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.