Alexion children's book series aims to help parents talk to kids about life with a rare disease

Alexion's "Inspired By" children's book series is designed to help families talk about rare diseases with their children. (Alexion)

When Rachel Pegram’s adult sons were younger, she often missed their soccer games and other school functions because of her generalized myasthenia gravis (gMG), a rare autoimmune disease that causes muscle weakness.

Although she explained her illness and treatment to her boys, now 19 and 21, she’s not sure how much they really grasped when they were little. 

Now, with her three-year-old daughter Grace nearing school age, she wanted an easier way to explain her condition—using words and pictures that could help a preschooler understand a “big adult issue.” 

So she worked with AstraZeneca’s rare disease unit Alexion Pharmaceuticals, which makes her IV infusion treatment, to create "Klara’s Talent," an illustrated children’s book about a mommy koala, who has gMG, and her daughter Klara—characters inspired by Rachel and Grace. In the story, the mommy koala is too sick to go to her daughter's talent show, so Klara brings the show to her instead.

“I think it’s really going to help her understand, even more than my boys understood when they were her age,” said the 47-year-old from North Carolina. “We read it a few times a week, especially if I’m getting ready to have a treatment.”

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The book, published in June for myasthenia gravis awareness month, was the first installment in Alexion’s new “Inspired By” children's book series, which is based on real patients and aims to help families affected by rare diseases.

Alexion released the second and newest book in the series, “A Day With Nurse Jen,” in late September. It follows real-life pediatric nurse Jennifer Lamothe, who treats children and teens with atypical hemolytic uremic syndrome (aHUS). Alexion is already exploring a third book and hopes to continue the series over time.

While the series is unbranded, Alexion’s C5 inhibitor Soliris has long been the standard treatment for aHUS, a rare disease that can lead to kidney failure, and its successor Ultomiris won an FDA nod for the indication in 2019. The FDA approved Soliris to treat gMG in 2017, and the company is eyeing a similar gMG nod for Ultomiris after a successful phase 3 gMG trial last summer. Combined, the two blockbusters brought in more than $5 billion in 2020 for Alexion, which AstraZeneca acquired for $39 billion in July.

“In listening to our patients, we recognized that they needed a way to talk to their children about these diseases in an approachable and age-appropriate way,” Mary-Sheila Leese, Alexion’s vice president of patient services and strategy, said in an email interview. “Our aim was to create a support tool for those conversations that would be informative while also normalizing life in a family affected by a rare condition.” 

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The plot and images in both books are drawn directly from patients’ experiences, said Leese. For example, "Klara’s Talent" addresses the guilt many parents feel when their disease prevents them from playing or attending a school function. 

In the second book, Nurse Jen meets children like 10-year-old Ben, who fears leaving the hospital in case he gets sick again, and 14-year-old Nicole, who worries about whether she’ll be healthy enough to try out for the school musical.

Leese said grandparents have read the books to their grandchildren, and children have shared them with siblings or cousins to help explain their experiences.

“Story time is an important part of family time,” Leese said. “But it isn’t often that those affected by rare diseases see their experiences on the page.”

Alexion is distributing the book through patient advocacy groups and through its patient support program OneSource.

Editor's Note: This story has been updated to remove the mention that nurse Jennifer Lamothe has aHUS, as she does not.