02/25/2026
When the FDA Moves the Goalposts, Children with Rare Diseases Lose by Jessica Haywood.
"For families with children who have Sanfilippo syndrome, a terminal type of childhood dementia, time is not measured in years or presidential cycles. It is measured in lost words, hospital visits, seizures and skills slipping away. Birthdays are bittersweet because we know there will be far too few of them.
"This community is not alone. Children suffering from thousands of rare and debilitating childhood diseases, such as Hunter syndrome, Duchenne muscular dystrophy, and many others, are declining every day.
"However, they don’t have to. The Food and Drug Administration can move today to prioritize therapies that have made a measurable difference for these children, such as the Sanfilippo type A gene therapy UX111, which has been helping patients for almost 10 years.
"My niece, Sadie, is almost 10 years old and is full of life despite her struggles with Sanfilippo syndrome. Sadie was diagnosed early, at only 3 months old. Around the same time, the first child was dosed with UX111. We have been watching this drug’s development for Sadie’s entire life. We have longed for it to reach approval so she could receive it.
"Unfortunately, the application has been delayed twice due to manufacturing documentation, not safety or efficacy concerns. For diseases with a life expectancy of 15 years, accelerated approval and regulatory flexibility must operate as intended.
"We’ve spoken to parents of children who have been treated with UX111 at varying ages. Their kids are running when they should be wheelchair bound. They’re eating by mouth when otherwise they should have a feeding tube. After seeing such promise, the FDA’s rejection last summer was devastating. And we weren’t alone, as many rare degenerative disease patients watched approvals for diseases like Duchenne and Spinocerebellar ataxia slow or come to a screeching regulatory halt. ..."
For families with children who have Sanfilippo syndrome, a terminal type of childhood dementia, time is not measured in years or presidential cycles. It