Why is there no cure or approved treatment for NKH?

There are a few reasons nonketotic hyperglycinemia has gone so long without a treatment that addresses its cause. The first is rarity. With an estimated 1 in 76,000 people affected, there are too few patients to attract the large investments drug development usually requires.

The second is biology. NKH does its damage in the brain, and getting a medicine to the right cells in the brain, at the right dose, is one of the hardest problems in medicine. Lowering glycine in the blood doesn't fully protect the brain.

The third is time. Many children with the severe form are critically ill as newborns, which makes research and clinical trials difficult and urgent at once.

So families have stepped up. Since 2017, the Drake Rayden Foundation has helped fund NKH research, including a gene therapy project launched in 2019. Tarah's conviction has never wavered: "Rare should not mean disposable. Rare should not mean not valuable."

Now, a one-dose gene therapy trial is planned for late 2026, and the remaining funding is the last hurdle. Your gift or your share helps get Drake and Vivian there.

Sources: MedlinePlus Genetics: Nonketotic hyperglycinemia · NORD: Nonketotic Hyperglycinemia