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.
Daniel S. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013 to provide strategic communications to life sciences companies. He is host of The Bio Report and RARECast podcasts, a senior fellow at the Center for Medicine in the Public Interest, and a member of the advisory board of the California Biotechnology Council.
How do you think the studies of natural history will affect long-term research for rare disease?