MINNEAPOLIS (WCCO) — A Minnetonka toddler and her family got the incredible news that the “miracle drug” keeping her alive won’t be discontinued after all.
CBS Minnesota shared Emma Peterka’s story in March. She has Niemann-Pick Type C, a progressive neuro-degenerative disorder that the Peterkas liken to Alzheimer’s, ALS and Parkinson’s — all rolled into one. Mitch Peterka, Emma’s father, says there are about 500 cases in the world. He and his wife Sara found out in January the drug that gave Emma her strength back, Adrabetadex, was being discontinued in October. Sara says the drug has put the light back in Emma’s eyes.
“She’s making more noises and she tries singing along to some of her favorite songs, which is really fun,” Sara said.
With the announcement of the discontinuation, the Peterkas felt abandoned. Then, two weeks ago, the manufacturer announced it found a new company to take on Adrabetadex. They plan to produce enough supply of the drug to last up to two years.
“The kind of feeling that you just want to run outside and just scream at the top of your lungs with excitement,” Mitch said. “To go from not having any hope because it was ripped away from you, to suddenly having that hope restored was incredible.”
The Peterkas have become close with several NPC families, including Denise Miller and her family from Phoenix, Arizona. Her son Woodrow started his Adrabetadex treatment last week, a couple months shy of his second birthday.
“It will give him a lot of years,” Denise said. “It will give him the ability to grow and develop like a normal child should.”
Denise believes the NPC community had strength in numbers in securing this significant drug extension. “We’re helping to save each other’s children’s lives,” she said.
There’s no cure yet, but signs are good that Emma will continue to break her own records of how long she’s able to stand up for. “The fact that this signals a path forward is just amazing,” Sara said.
Every two weeks, the Peterkas drive to Chicago to receive the drug. The Millers will now be flying there from Phoenix twice a month. One of the many questions both families still have is whether the drug will be more readily available closer to home.