GREENWOOD, Mo. -- For the first month of her life, Brooklynn Enos seemed to be a healthy, typical baby girl.
“Completely normal. Kicking and screaming, smiling, started breastfeeding immediately," her mom, Rachel Enos, said. "Just a normal baby. Had no idea at all that we were in for something special."
Something special and something quite alarming. Bows and nail polish were suddenly joined by breathing machines and feeding tubes. Brooklynn`s young life took a drastic turn weeks after she was born.
Tests that showed Brooklynn had Spinal Muscular Atrophy 1, or SMA-1.
“She ended up losing her swallow, having an NG tube put down her nose, losing almost all of her movements in all of her limbs, her legs her arms, she couldn`t move her head, her smile was weakening, and we thought we were losing her,” Enos said.
A few weeks later, Brooklynn got pneumonia, that coupled with her dwindling strength, gave doctors reason to believe she would be another SMA casualty.
“That`s pretty much all they said," Enos said. "They didn`t tell us anything else besides nine to twelve months would be a great timeline for her life."
The life expectancy for SMA-1 is often 18 months at most. Babies born with the disease lose their ability to move their arms and legs, becoming paralyzed, they also lose the ability to swallow and can`t breathe on their own. All of this happens while their brain continues to function normally.
What happened with Brooklynn is different.
“She can smile, she`s starting to laugh,” Enos said.
On Oct. 5, Brooklynn did what her doctors didn't expect her to do. She celebrated her first birthday.
Rachel and Matthew say one thing has made the difference between slowly watching their little girl slip away and making big plans for the future.
“It`s a miracle drug," her mom told Fox 4. "We`re watching a miracle right before our eyes. This drug is life changing."
It`s a new drug called Spinraza, and Brooklynn is one of the youngest patients ever to try it. Dr. Anne Connolly is at the helm of a research study testing the effects of Spinraza . She says studies show Spinraza helps children with SMA regain milestones like kicking and smiling, just like Brooklynn did.
“She`s definitely benefitting from the medicine,” Dr. Anne Connolly said.
Once every four months for the rest of her life, Brooklynn will go to children's hospital in St. Louis to get her Spinraza through a spinal tap.
It is extremely costly, however.
The cost has drawn wide criticism. It costs $750,000 for treatments in the first year, and it will cost $375,000 for the three treatments Brooklynn will need every year.
It's been a struggle for the Enos family. Rachel quit a full-time career to give Brooklynn the 24-hour care she needs.
“When you see this- I don`t care- I would live in a shoebox. You know what I mean?" she said.
Just two days after the most recent treatment, Rachel says Brooklynn has more strength in her arms and legs, and she's moving with noticeably less effort.
It`s results such as these that have researchers excited about the future of SMA treatments.
“This is a very hopeful time. This is the first big breakthrough, Spinraza is the first big breakthrough," Dr. Connolly said.
Another breakthrough is Senate Bill 50. It was signed into law this year- it makes Missouri the first and only state so far to screen all newborns for SMA.
“If you just look at an infant, you can not tell. The physical exam at birth does not show that they`re becoming weaker, but by two to three months, they are profoundly weak,” said Dr. Connolly.
Screening will begin in 2019, and it could save the lives of children born with SMA by getting them access to treatment right away.
"I`m not putting any limits on her, I`m not putting a date on her, I`m not saying like that because no- you`re going to be here forever, you`re going to outlive me for sure,” Enos said.
Fox 4 asked a Biogen spokesperson why the drug cost so much.
She responded in part:
"It strived to achieve an appropriate balance among three key pricing principles - clinical value, impact to the healthcare system, and commitment to patients and advancing science through the funding of research and development."
Biogen also offers a program that aims to make Spinraza more affordable for families.
If you want to follow Brooklynn's fight and the Enos family adventures as Brooklynn continues to amaze doctors, you can like their Believe in Brooklynn Facebook Page.