Rare disease clinical trials are constrained 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. https://safeersappliancers.shop/product-category/kenwood-ck307g-90-cm-dual-fuel-range-cooker-black/