Why Natural History Studies Matter to Rare Disease Patients

Peroxisomal disorders are a group of rare and heterogeneous metabolic diseases. Earlier this year, the National Organization for Rare Disorders selected the Global Foundation for Peroxisomal Disorders as one of 20 rare disease groups to undertake a natural history study with support from the U.S. Food and Drug Administration. We spoke to Melissa Bryce Gamble, president and co-founder of the Global Foundation for Peroxisomal Disorders, about the natural history study, why such studies are critical for expanding the understanding of a rare disease, and how they help inform the development of therapies.