Expert Rare Disease Care at Gillette Children’s

Shortly after birth, Annika’s mother, Anna, and her grandmother, 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.”

When she was 9 months old, Annika was diagnosed with spinal muscular atrophy (SMA), a muscular disease that affects the motor nerve cells in the spinal cord and gradually takes away a child’s strength. Being diagnosed with the most severe form of the disease, type 1, Anna prayed Gillette Children’s pediatric neurologist, Randal Richardson, MD would select Annika to participate in the clinical trial and research into Spinraza ™, a drug used to treat SMA.

Anna Hendricksen wanted this more than anything for her daughter.

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 as an “Embrace” clinical trial site.

Annika began regularly coming to Gillette to see Dr. Richardson to receive Spinraza treatments.

Rare diseases are common at Gillette

Gillette is known worldwide as an expert in rare disease care. “Rare diseases are actually common at Gillette,” says Arthur Beisang, MD, complex care pediatrician and 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 work with respected medical institutions around the world to improve outcomes. Partnered with clinicians, they do groundbreaking work.

Newborn screenings bring hope

SMA is the most common genetic cause of infant death, affecting 1 in 11,000 infants annually. Thanks, in part, to research at Gillette, all babies born in Minnesota are now screened for SMA unless their parents opt out of newborn screenings. This is important because starting treatment as soon as possible can help curb symptoms.

“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,” Minnesota Health Commissioner, Jan Malcolm, says.

Thriving with SMA

Today, Annika is in the second grade and 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 to see.”

Thanks to her treatments, Annika is now strong enough to travel. She’s been to Disney World and recently spent time at Fragrant Isle Lavender Farm in Wisconsin. “Annika loved lying in the fields, seeing the butterflies, and smelling the lavender,” Anna says.

One of the most important things

“I very openly tell people that this work on SMA is probably the most important project of my life. It’s one of the most important things I’ll ever be involved with,” says Dr. Richardson.

Anna Hendricksen agrees and says, “We’re hopeful for Annika’s future. We’re so grateful to Gillette, Dr. Richardson, and the team. They’ve all worked to help our family and to make sure Annika can be her best.”

Grandmother Deisy also marvels at what she considers one of the most significant improvements. “Annika’s smile is back!”