SIMPATHIC is a Horizon Europe project aiming for the acceleration of drug repurposing for rare neurological, neurometabolic and neuromuscular disorders. Important accelerating factors for SIMPATHIC are early involvement of all stakeholders, including patient organisations, industry and regulators; a direct step from iPSC-derived cell models to in-human studies; parallel drug screens in cells from patients with different disorders but overlapping molecular signatures and clinical symptoms; and basket trials including patients with different disorders but overlapping clinical symptoms.