I feel that a small biotech may be ideal for developing a new drug for a rare disease like Wolfram syndrome. A small biotech has a narrow focus, which may be suitable for creating a drug targeting a specific pathway and a specific disease. Mr. John Crowley and Dr. William Canfied (trained in Internal Medicine at Washington University) took this strategy and created a novel drug for Pompe Disease. They started a company in 2000, and Mr. Crowley's children with Pompe disease received their drug in 2003. Their small biotech was eventually acquired by Genzyme.