Steven Ringel

On February 23, the FDA did something the rare disease community has spent a decade fighting for: it issued the Plausible Mechanism Framework, a formal pathway for individualized therapies where traditional clinical trial evidence standards are not appropriate. Sponsors can now meet the evidence standard through biological proof of concept. Identify the genetic problem, show the therapy targets it, demonstrate it works at the molecular level, and you can dose an initial cohort of patients.

William King Engel MD died peacefully at his home in Ann Arbor, MI, at the age of 94. Born in St. Louis, MO, Dr. Engel graduated from Johns Hopkins University, earned his medical degree from McGill University and completed his neurology residency at University of Michigan and the newly formed National Institute of Neurological Disease and Blindness (NINDB) at NIH.