Posted by Michael Wonder on 05 Jul 2018
A risky drug approval lesson
4 July 2018 - What the FDA can learn from progress against muscular dystrophy.
Some welcome news arrived recently for boys with a condition called Duchenne muscular dystrophy. A new therapy is showing promising results, and it raises a question for the FDA: what if society had missed this moment?
In late June Sarepta Therapeutics, a biotech company, released data on a gene therapy for Duchenne, a disease that weakens muscles and organ function. Patients tend to die before age 25.
