“When life gives you lemons, make lemon gelato!” Brent is disabled by PKAN, a rare neurodegenerative disease, yet supported by his family he is making many of his dreams come true.
Description
Veronica joins David Rintell, Head of Patient Advocacy at BridgeBio to tell her son Brent’s story. When Brent was nine, his family discovered that he had a rare genetic disease called Pantothenate Kinase-Associated Neurodegeneration (PKAN), a condition without any available treatments. Veronica shares how she and her family have supported Brent as he has stubbornly and victoriously succeeded in giving his valedictorian speech in high school, attended community college, and started a local gelato business.
Mallory Harden, Ph.D., Director of Business and Operations at CoA Therapeutics, a BridgeBio company, provides an overview of PKAN. She explains how PKAN is a progressive and fatal neurodegenerative disease which is often diagnosed in childhood. As it progresses, people are faced with uncontrolled movements, walking and balance challenges, and cognitive changes.
United States
