Comment from Cure Sanfilippo Foundation

Summary: The Cure Sanfilippo Foundation, a nonprofit patient advocacy organization, supports the proposed Plausible Mechanism Framework as a significant step forward for developing therapies for ultra-rare diseases. They request specific clarifications to ensure the framework is inclusive of various therapeutic modalities, uses "fit-for-purpose" natural history data, allows for flexible endpoints, and adopts "right-sized" manufacturing requirements for small patient populations.