Glycogen Storage Diseases

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.
During his address to Congress this week President Trump shone a spotlight on Megan Crowley, a 20-year-old Notre Dame sophomore with Pompe Disease, which is caused by mutations in the GAA gene.