26 January 2018 - The economist behind the ‘priority review voucher,’ which advances treatments for neglected diseases.

You’ve probably never heard of biallelic RPE65 mutation—and count yourself lucky. It’s a genetic defect that causes a form of retinal dystrophy. People born with it gradually lose their vision, often while still children, until they go totally blind. 

But they are now fortunate: In December the FDA approved a new gene therapy called Luxturna that corrects the mutation and reverses the course of the disease.

Read Wall Street Journal article