Joshua Burns

PXT3003: Lessons for therapeutic development in Charcot-Marie-Tooth disease type 1A and related neuropathies

Summary Most drug development stories end predictably: promising compounds either succeed or fail. Occasionally, however, a therapy repeatedly returns from apparent extinction. PXT3003 for Charcot-Marie-Tooth disease type 1A (CMT1A) represents one such example. More than a decade after early promise, the program has su...