Inspired by a true story, the movie Extraordinary Measures features Brendan Fraser as a father on a mission to find a cure for a rare, genetic disorder called Pompe disease that is killing his two youngest children. Harrison Ford plays the scientist the father teams up with.

At the movie’s opening Friday, Jan. 22, at the Regal Short Pump, Krystal Hayes and daughter Haley, 4, who has the disease, will provide information on the condition. They will have a table set up at about 6 p.m. near the movie entrance.

Hayes, who lives in Bracey, Va., with her family, is daughter of Arlene McKenzie of Henrico County.

Ms. Hayes talked about the disease and how it has affected Haley and their family.

Q. What is Pompe disease? How is it pronounced?

Krystal Hayes: Most people say \Pom-pey\. I have heard \Pomp\.

It is a rare, genetic disease that affects the muscles. The body is missing an enzyme that breaks down glycogen. The glycogen gets stored in all the muscles throughout the body and kind of make the muscles deteriorate.

There are different types – infant type and adult type. With the infant type, they have severe cardiac involvement, heart involvement. That is where the infant type is more severe because the heart becomes very large and functions very poorly. Adults usually don’t have that heart involvement.

Q. When was Haley diagnosed?

Krystal Hayes: Haley was diagnosed at 6 months old. We were sent to (VCU Medical Center) because we had taken some lab work that came back with some of the levels elevated. She wasn’t gaining much weight. We kept waiting for a month or two to see if she would get better. Once we got to MCV, about two weeks later we got the actual diagnosis. They found her heart issues first, a few days after we got there, and from there, why does she have a heart issue.

Q. What is treatment?

Krystal Hayes: There is a treatment called Myozyme. That is an infusion. Most people get it every two weeks. Haley actually gets it every week because she was found to do really well when she got more of the medicine. She has a port-o-cath.

It basically just slows down the progression of the disease. It works differently for every person who I have ever talked to who takes it. Some kids do really well. Some kids have a reaction and can’t take it at all. It really is a case-by-case thing. Haley does pretty well on it. She just turned 4, which is amazing. If you have infantile-onset, they tell you without treatment you don’t live past the age of one because of the cardiac involvement. Her heart is now normal. Any muscle that was affected in the beginning doesn’t fix itself. You can’t reverse that.

Q.Where does she get the infusion?

Krystal Hayes: She now goes to Duke. We went to (VCU) for a little while and we stayed up there for a month while she was being diagnosed. Haley was their only case of this.

We live between the two, so we just went down to Duke. They see so many kids with this we felt more comfortable.

Q. Do they have a special center for this disorder?
Krystal Hayes: They just have a very big genetics team. This medicine she gets, Myozyme, was founded at Duke, from some of the doctors at Duke.

Q. Does insurance cover the treatment?

Krystal Hayes: We have had some issues over the few years. Right now, it’s covering it. However, she is about to reach her lifetime maximum because the medicine is so expensive. Her treatments each week are about $18,000. We are OK until we reach the lifetime maximum, which will actually be in a couple of months. We are trying to research different alternatives of what to do at this point.

Q. Is there a national organization for this disease?

Krystal Hayes: There are a couple of different organizations. There is United Pompe Foundation. There is the AMDA, which stands for Acid Maltase Deficiency Association, because that is another name for it. There are some worldwide organizations and foundations.

Q. What are you planning to do at the movie theater?

Krystal Hayes: Basically what we are trying to do is promote awareness of the disease, to have Haley there so people can actually put a real face with the name and the movie. We are going to be passing out brochures and cards and pamphlets, things like that, so people can get more information about Haley or Pompe disease.

Q. What do you know about the movie?

Krystal Hayes: It’s based on a true story about a father who has two children with Pompe disease before any treatment was available. He kind of left his really nice job to start his own company to try to find a treatment for this disease. It shows his journey along the way to get a medicine approved for treating the disease.

Q. Is that what led to Myozyme?

Krystal Hayes: It’s really honestly debatable among the whole Pompe community. He really didn’t find a medicine. He maybe helped it come quicker. To me it’s about his journey. He has a great story.

Family photos courtesy of Krystal Hayes