Anna Hendricksen wanted this for her daughter more than anything she dared wished for.
Anna prayed Gillette Children’s pediatric neurologist, Randal Richardson, MD would select her daughter, Annika, to participate in the clinical trial and research into Spinraza™, also called nusinersen, a drug to treat spinal muscular atrophy (SMA). It is a muscular disease that affects the motor nerve cells in the spinal cord and gradually takes away a child’s strength. SMA is the most common genetic cause of infant death, affecting 1 in 11,000 infants annually.
Shortly after Annika’s birth Anna and her mother, Deisy, noticed she was missing milestones and declining. “One day I looked at my granddaughter and she lost the ability to even smile,” Deisy recalls. “To me, it seemed like she was melting away before our eyes.” Annika was diagnosed with spinal muscular atrophy type 1, the most severe form of the disease.
In March of 2016, the Hendricksen family traveled from Green Bay, Wisconsin and began participating in the “Embrace” clinical trial at Gillette. After a rigorous inspection process, the drug company, Biogen, and the Food and Drug Administration approved Gillette Children’s as an “Embrace” clinical trial site.
Rare diseases are common at Gillette
Gillette Children’s is known throughout the world as an expert in rare disease care. “Rare diseases are actually common at Gillette,” says Gillette Children's complex care pediatrician, Arthur Beisang, MD. He’s also the co-director of the Gillette Children’s Rett Syndrome Center of Excellence. Beisang adds, “We’re really set up to manage a lot of health issues that come with rare diseases and we’re able to coordinate excellent care for these children.”
Since its founding in 1897, Gillette has been at the forefront of treating children who have conditions affecting the brain, bones, and movement. Medical research is a key part of Gillette’s mission. Through clinical trials, the Gillette team of clinical scientists works with respected medical institutions around the world to improve outcomes.
Gillette has nine clinical scientists on staff. Research director, Joyce Trost, says it’s a strong team. “We have nine PhDs and all they do is research. These clinical scientists are partnering with the clinicians to do some groundbreaking work.”
Newborn screenings for SMA brings hope
Thanks, in part, to research at Gillette Children’s, all babies born in Minnesota are now screened for spinal muscular atrophy (SMA) unless their parents opt out of newborn screenings. This is important because starting treatment as soon as possible can help curb the affects of SMA.
"Adding SMA to the state's newborn screening panel is an important move that will help save families from the heartbreak of losing a child or losing precious time when treating their child that could result in better outcomes," former Minnesota Health Commissioner, Jan Malcom, says. "This addition is the result of hard work by many people," Malcom adds.
Annika's smile is back
Annika regularly comes to Gillette Children’s to see Dr. Richardson and receive Spinraza treatments.
Today, Annika is in the second grade and is learning to read. Through intense therapy, she also learned how to use an eye-gaze communication device.
“Annika has a special device that looks like an iPad,” Anna says. “She uses her eyes to look at words and symbols on the screen so she can communicate. It has made a world of difference to be able to understand what my daughter wants. I even enjoy it when she’s a little bossy and tells me exactly which Disney movie she wants see.”
Thanks to her treatments, Annika is now strong enough to travel. She’s been to Disney World and recently she spent time at Fragrant Isle Lavender Farm in Wisconsin. “Annika loved lying in the fields, seeing the butterflies, and smelling the lavender,” Anna says.
Grandmother Deisy also marvels at what she considers one of the most significant improvements. “Annika’s smile is back!”
One of the most important things
“I very openly tell people that this is probably the most important project of my life. It’s one of the most important things I’ll ever be involved with,” says Gillette neurologist Randal Richardson, MD.
Anna Hendricksen agrees with Richardson and says, “We’re hopeful for Annika’s future. We’re so grateful to Gillette, Dr. Richardson, and his team. They’ve all worked to help our family and to make sure Annika can be her best.”