ESCONDIDO — It was a devastating diagnosis with a fatal outcome.
But for one Escondido family, their fight to prolong their son’s life is one of patience, frustration, anger and honesty.
Todd and Angie Morrow, however, are currently in a holding pattern as a pharmaceutical company and the Federal Drug Administration (FDA) battle over the fate of critical drug.
In the meantime, the two are caring for their son, Ryan, a spry 6-year-old with a beaming smile as he wages a fight against a rare disease known as Duchenne muscular dystrophy (DMD).
Affecting mainly boys, most diagnosed die in their late teens to mid-20s due to heart or lung failure, according to the Muscular Dystrophy website. In rare cases, patients live into their 40s and 50s. There is no cure or medications to treat DMD.
Symptoms typically begin between the ages of 3 and 5, and affect one in 3,500 boys.
“You don’t really know what’s behind the disease,” Todd Morrow said. “There’s three major mutations. He has a stop codon. Each drug that is being developed is designed for each type of defect.”
The Morrows lives were changed forever about four years ago when they noticed Ryan wasn’t walking as soon as he should. After several doctor’s visits, nothing was discovered, and Ryan began to walk.
However, Todd Morrow still felt something was amiss. They turned to a neurologist who performed a blood test revealing the DMD diagnosis when Ryan was about 18 months old. When revealed there was no cure, the new parents were crushed.
The struggle, though, is Ryan was not allowed into two clinical trials for the drug Translarna, which is manufactured by PTC Therapeutics. The first trial after Ryan’s birth was when he was 3, too young. The second came several months ago, but he was too old at 6. The cutoff was at 5.
The Morrow’s research and conversations with doctors and other patients told them the drug was safe and prolonged life. But being too young, then too old, Ryan’s best option now is taking Deflazacort, which he does.
“We didn’t even know what it was,” Todd Morrow said of the initial diagnosis. “Only three drugs really work on dystrophin. He’s in a honeymoon right now. He’s putting on muscle faster than it is degrading. At about age 10, it peaks and it is a rapid decline.”
Translarna has been approved for conditional use in Europe, he added, but it has stalled through the approval process in the U.S.
He said the drug is a “game changer” and would slow the muscle loss and reduce the affects of the disease.
The Morrows have scoured the country for the best doctors and hospitals to treat Ryan. At first, they saw doctors in San Diego, but discovered one of the top facilities in the country at the University of California, Davis, about 500 miles north near Sacramento.
Last year, UCLA opened a facility, which the Morrows now visit and reduces the stress of days away from home for Ryan.
“People really want to help,” Todd Morrow said. “We are just looking for the best care for Ryan. I can’t even explain the day you find out, you find out it’s a fatal disease and you can’t get to the option. It’s the most important thing to you.”
DMD is a genetic disease causing muscle weakness and strips patients of their ability to walk, move their arms and other motor functions. The disease is caused by nonsense mutation passed through the X chromosome from the female to the male.
Since the male only has one X chromosome, the end result is fatal.
There is a clinical trial, but Ryan is ineligible. In February, the FDA refused to review clinical data for the drug, while PTC Therapeutics announced it would appeal the decision.
“The drug had in trail quite awhile (11 years),” Morrow said. “It was in Phase 3. It’s safe, effective, but they were confirming it.”
Despite Ryan’s prognosis, he began kindergarten this week and said he was excited to start school.
His parents, meanwhile, said the school has been great with its assistance and acceptance to learn about their son’s condition.
Ryan, though, does ask his parents why he struggles to keep pace with his friends when they play or run. Todd and Angie Morrow, though, are honest with him.
Although Ryan Morrow is only 6, he hasn’t fully grasped his condition, but his parents said they will always be honest with their son about his disease.