Developing therapeutics for rare diseases comes with unique challenges—small patient populations, high variability in treatment response, and the lack of conventional statistical and translational approaches. This is where biosimulation and quantitative systems pharmacology (QSP) play a game-changing role.
In this expert video, Piet van der Graaf, PharmD, PhD, explains how biosimulation is reshaping rare disease drug development, using a powerful example from our work with Sanofi on Pompe Disease. This innovative approach is accelerating development, reducing risks, and ultimately bringing life-changing therapies to patients faster.
