Like other nonprofits, the Spinal Muscular Atrophy Foundation has provided a rich source of funds for drug developers working to treat a rare condition. PTC Therapeutics and Isis Pharmaceuticals have both been bankrolled by the foundation for preclinical work in the field. And now Novartis is collaborating with the foundation with plans to launch a study in 2013.
But unlike many other nonprofits, this foundation is largely backed by one man: the wealthy hedge fund operator Dinakar Singh.
Singh has provided $100 million of his own money to help find new therapies for spinal muscular atrophy, spurred by his intense desire to help his daughter, who suffers from the malady. And in a lengthy profile in Bloomberg Markets magazine, investigators say Singh's money and business savvy has revved up their work, moving the field from a scientific backwater to a "high-speed initiative."
"I was fearful and anxious that treatments would be developed, but far too late to save Arya," Singh tells Bloomberg. "We didn't want to find out 25 years later that the science was really there but there isn't a drug because nobody focused on it."
Singh says he plans to enroll his 11-year-old daughter in one of the first human studies of an experimental therapy.
- here's the article from Bloomberg