Hello from San Diego, where I always happen to be, but where this weekend I had the delight of hanging out with coworkers including Adam Feuerstein, Jonathan Wosen, and Angus Chen, along with others who are here for this year’s meeting of the American Society of Hematology. If you haven’t done so already, it’s not too late to sign up for STAT’s handy ASH newsletter here.
One dad’s quest to develop rare disease treatments
Terry Pirovolakis successfully developed a gene therapy for his son, Michael, who has an ultra-rare disease called SPG50. Now, he’s offering hope for other families facing similar hurdles through his company, Elpida Therapeutics, which is seeking to develop treatments for diseases too rare for pharmaceutical companies to tackle.
Although Pirovolakis has helped several children gain access to groundbreaking therapies, STAT’s Jason Mast writes, systemic funding shortages and the complexities of scaling his model underscore how difficult it remains to address rare disease.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
