CureDRPLA

CureDRPLA is a U.S.-based nonprofit rare-disease organization focused on accelerating research and treatment development for dentatorubral-pallidoluysian atrophy (DRPLA), an ultra-rare neurodegenerative disorder caused by pathogenic expansion in ATN1. Founded in 2019 by Paul and Andrea Compton after their son was diagnosed with DRPLA, the organization connects patients, families, clinicians, and researchers while funding disease-focused preclinical research. Its activities include development of a global patient registry, support for a natural history and biomarkers study, and sponsorship of multiple therapeutic research efforts aimed at lowering mutant ATN1 expression. CureDRPLA appears to function primarily as a nonprofit research catalyst and patient-advocacy organization rather than a traditional venture-backed biotech operating its own commercial drug pipeline.