1 in 12 people has an orphan disease. 95% of orphan diseases have no treatment.
Each disease is caused
by a spectrum of genetic mutations.
Perlstein Lab studies each mutation
in model organisms to produce precision medicine.
Leave no mutation behind
PLab is the first Public Benefit Corporation accelerating precision drug discovery for 5,000+ rare genetic diseases.
It has been a while since you have heard from me. We are deep in post screen characterization of our NPC hits so I thought I would take a step back to the beginning to discuss NPC disease biology. We focused a bit on the cholesterol and lipid metabolism aspects of Niemann Pick C disease […]