BAYESIAN MODELING AND SIMULATION TO INFORM RARE DISEASE DRUG DEVELOPMENT EARLY DECISION-MAKING: APPLICATION TO DUCHENNE MUSCULAR DYSTROPHY.

Bayesian modeling and simulation to inform rare disease drug development early decision-making: Application to Duchenne muscular dystrophy.

Rare disease clinical trials are constrained Hair Accessories to small sample sizes and may lack placebo-control, leading to challenges in drug development.This paper proposes a Bayesian model-based framework for early go/no-go decision making in rare disease drug development, using Duchenne muscular dystrophy (DMD) as an example.Early go/no-go dec

read more