Is gene therapy a cure for NKH?

It's the question everyone asks, and it deserves an honest answer. Gene therapy aims to fix the root cause of NKH rather than manage symptoms, which is why the family calls EmetAbba potentially curative. But no gene therapy for NKH has ever been tested in people. Drake and Vivian's trial will be the first.

First-in-human trials exist to learn. Doctors will watch closely for safety and for signs that glycine levels and symptoms improve. It's also important to remember that a working gene may not undo injuries that have already happened in the brain. Even a good result might look different from what anyone imagines.

The family holds both truths. They are full of hope, and they know there are no guarantees. Tarah has written: "Drake and Vivian's future have always been uncertain, but our faith in the God who holds their future is steadfast."

What is certain is that without funding, the trial can't happen at all. The family is asking people to help give Drake and Vivian the chance. Give toward the trial or share their story.

Sources: U.S. FDA: What is Gene Therapy? · Saving Drake and Vivian (GoFundMe updates)