Comment from Adult Polyglucosan Body Disease (APBD) Research Foundation

Summary: The Adult Polyglucosan Body (APBD) Research Foundation supports the FDA's draft guidance on the Plausible Mechanism Framework for individualized therapies for ultra-rare genetic conditions. They advocate for further clarifications regarding natural history data, transition pathways from n-of-1 studies, and expanded funding and manufacturing exemptions to accelerate treatment for patients with GSD IV.