A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer than than 6,000 people.

“This new approval demonstrates our commitment to approving advancements in treatment that will give patients living with PKU different options for care.” PKU affects about 1 in 10,000 to ...

There are only around 17,000 people living with PKU in the US, and 150,000 globally, but - despite that - treatments can command healthy sales. Meanwhile, the positive results in PKU are a boost ...

Untreated PKU can result in global developmental delay or severe irreversible intellectual disability, as well as growth failure, hypopigmentation, motor deficits, ataxia and seizures. Living with ...