Rivulet Media Inc (fka RIVU)

[CPAP]

Hate to burst the bubble (I'm in BMSN right now), but the disease is so rare that it would be hard to get the sample sizes you describe in phase II or III less than 1 in a million people get this disease and those that do die within two years. You would need all diagnosed cases included appropriately in each case of the trials. Not saying they shouldn't try, but its not as easy to do with a very rare disease.

Journal article on aplastic anemia from 2008:
http://www.haematologica.org/content/93/4/518.full

Vital Stats from ACS on it:
http://www.cancer.org/cancer/aplasticanemia/detailedguide/aplastic-anemia-key-statistics

The problem is there is such a small subset of the population from which you are to develop a sample.

Hope it pans out for BMSN though, since I picked up a whole bunch last week!