The presentation will outline where there are opportunities/challenges to implement translational models for rare diseases in drug discovery. My presentation will provide examples of where translational models have been utilized in rare disease modeling and pharmacology to progress molecules in early drug discovery. This will include 1) neuromuscular junction model to investigate functional impairment induced by AChR+ myasthenia gravis patient sera.; 2) peripheral neuropathy models to investigate inhibiting complement activation and 3) Cardiomyocyte models for myotonic dystrophy. For each of the examples I'll conclude with key findings and lessons learned.