MN documentary chronicles groundbreaking treatment for rare genetic disease

A rare genetic disease and the new life-saving treatment being offered in Minnesota is the focus of a new documentary that premiered in Minneapolis on Monday.

"Sequencing Hope" follows a family from Alabama that comes to M Health Fairview Masonic Children's Hospital in order to try and save their daughter’s life. Celia Grace Hamlett was diagnosed with a rare disease called Metachromatic Leukodystrophy (MLD). It’s a fatal disease that often kills patients within a few years.

"Knowing that I was going to lose her was just devastating, and I didn’t know how to handle it," said Celia Grace’s father, Gary Hamlett.

The documentary, directed by a pair of Twin Cities filmmakers, follows the Hamlett family as Celia Grace becomes the first patient in the United States to receive innovative gene therapy at the University of Minnesota.

"When the doctor called and said your daughter is going to make history books… I was just overwhelmed with joy," said mom Kassie Hamlett.

MLD attacks the central nervous system, leading to the loss of ability to walk, talk, and eventually puts the patient in a vegetative state. Many patients don’t live past 7-years-old.

Celia Grace is now two years out of treatment, and shows no signs of disease.

Since the documentary was filmed, the treatment received FDA approval and now is the most expensive drug on the market with a wholesale price of $4.2 million.