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Rare Diseases and Orphan Diseases

Rare diseases affect a small percentage of the population, with Duchenne muscular dystrophy (DMD) and cystic fibrosis as examples. Orphan diseases, however, lack adequate treatment options regardless of prevalence. While most rare diseases are orphan diseases, some common conditions like malaria can be considered orphan due to low commercial interest. Both face limited research, diagnostic challenges, and treatment barriers, requiring greater investment in drug development.