These smaller population “Orphan Diseases,” defined as those that affect less than 200,000 people across the U.S., collectively impact 25 million Americans. This substantial public health issue warrants Lancet's call-to-action.
Inborn Errors of Metabolism
We recently discussed the impact of President Trump's address before Congress (and the world) on the rare Pompe disease. Now, we dive into the topic of why these types of “Orphan Diseases” are so important to understand and be aware of, as to help promote and encourage therapeutic and potentially-curative research.