Imagine being a child filled with an amazing zest for life but so physically limited that you have difficulty moving your arms and legs. You are confined to a wheelchair.
Imagine, too, having to overcome many bouts of pneumonia, a very difficult spine surgery and, because you sometimes have trouble swallowing, your lungs are compromised. You also receive much of your nutrition through a feeding tube because of the risk that many solid foods pose for respiratory complications.
Now imagine you have grown to be a teenager – 14 to be exact – and despite all your physical limitations, every kid in school wants to be around you. Not only are you a social butterfly, but you always see the positive in every situation. Sprinkle in an infectious smile, a little funky color in your hair, your very own “Glitter Party” YouTube channel and there is only one young woman to make this bit of imagination come true – Parker Herman.
SMA: A Diagnosis No Family Wants to Hear
Parker was born with a rare, incurable neuromuscular disease called SMA, or spinal muscular atrophy. Children with this disease cannot control their muscles because they lack an essential protein related to certain nerve cells, causing muscles to become very weak. These kids typically become very fatigued, and things most of us take for granted – such as walking, eating and even breathing — can be an incredible challenge.
For Parker, an 8th grader who lives about an hour’s drive from Madison in Juneau, Wisconsin, life is not easy, but with top-notch care from her UW Health medical team and lots of love from her single parent father, Matt, and grandparents Ellen and Dan, Parker has overcome many hurdles.
“No parent ever wants to be told that their child has SMA,” says Mary Schroth, MD, a pediatric pulmonologist who cared for Parker for nearly 10 years at UW Health’s American Family Children’s Hospital in Madison before becoming chief medical officer at Cure SMA in 2018.
When she began caring for kids with this disease more than 25 years ago, Dr. Schroth knew little about SMA. She has learned – working side by side with her patients and their families – how to make the lives of countless children better, and her reputation in the field has generated many patient referrals from hundreds of miles away.
“We could do much better.”
“Not that long ago,” says Dr. Schroth, “parents of newly diagnosed SMA babies were told to take them home and love them and that their lives will be short – usually less than 2 years,” says Dr. Schroth. “Understandably, these families found these words incredibly devastating. I thought we could do much better.”
Without question, the journey for hope has been sluggish. Until very recently, medical treatment for SMA patients – aside from supportive care – has proved elusive. Yet, as Parker shows us, a positive attitude can pay off.
Since being approved by the Federal Drug Administration in late 2016, the first-ever SMA therapy, known as Spinraza™ (nusinersen) has been improving the quality of lives for children like Parker.
“My energy has totally increased,” Parker says. “Before, I would be wiped out and totally exhausted after school. Now I can stay up until 8 pm with all the energy in the world.”
Even more promising, says Dr. Schroth, additional and potentially even more powerful SMA therapies are now in the pipeline. The key, she says, is beginning treatment as soon as SMA is diagnosed.
“Every day that a baby with SMA goes untreated means more loss of motor neurons,” says Dr. Schroth. “Fortunately, newborns are now starting to be tested for SMA at birth so that if a baby is diagnosed, treatment can start immediately, and we can hopefully limit the nerve and muscle damage. Several states have already begun testing babies for SMA at birth and within a few years, we think most newborns across the country will be tested.”
Parker Dreams Big
For Parker, who loves summer camp, video games and all things glittery, dreaming big is something wired into her very being. As for what she aspires to pursue later in life, anything from fashion design to genetics are on Parker’s list. She also finds great fulfillment in lifting the spirits of other kids with SMA and hanging out with her friends who “don’t see the wheelchair; they just see me.”
Stay positive, she tells others who have walked in her shoes, even though times can be hard.
“Technology is changing, and there are ways it might help,” she adds. Dr. Schroth agrees and finds Parker’s resilient outlook the perfect allegory for the promise of current and future SMA treatments.
“I wasn’t sure I would see these advances in my lifetime,” says Dr. Schroth. “I am also honored to learn so much from children like Parker. She is truly a light who shines brightly.”