Argonaut manufactures orphan drugs, including many personalized medicines for patients with rare conditions. Leading up to Rare Disease Day on February 28, we have been sharing patient stories to raise awareness and highlight the people at the heart of our work.
Amelia and Makenzie’s story
Amelia was diagnosed with juvenile Batten disease at 6 years old — a rare and fatal neurological disorder — after years of symptoms and searching for answers. Soon after, her twin sister, Makenzie, received the same devastating news.
Today, they are 15, turning 16 next month. While the road remains uncertain, their parents turned grief into action by founding ForeBatten to fight for a different future for their daughters and the Batten community.
That work has already led to a first-in-human RNA clinical trial tailored to their rare mutation and, for the first time since diagnosis, real signs of progress.
If you’d like to support the Batten community and the research bringing hope forward, you can learn more here: https://www.forebatten.org/
Layken’s story
Layken is a joyful little girl living with a rare HNRNPH2 neurodevelopmental disorder. It took years of appointments, testing, and persistence to finally get answers.
Today, Layken is nine and loves spending time with her sister, Londyn. Whether they’re swimming, playing doctor, or attending therapy sessions together, their bond is strong and their laughter fills the room.
She is receiving personalized treatment through the n-Lorem Foundation.
If you’d like to support patients like Layken, and the research making treatment possible, you can learn more here: https://www.nlorem.org/support/
Sonia's story
Sonia was 25 when her mother passed away from Fatal Familial Insomnia– a rare, inherited prion disease. Shortly after, she learned she carried the same mutation.
Instead of stepping away, Sonia and her husband changed the course of their lives. They went back to school, earned Ph.D.s, and dedicated their work to developing a treatment for prion disease.
Today, that research is in clinical study.
If you’d like to support their work, consider making a tax-deductible donation to Prion Alliance – a 501(c)(3) nonprofit Soni and Eric created to fund research: https://www.cureffi.org/about/
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Anasi's story
Anais is 9 years old and lives with a rare HNRNPH2 genetic disorder. It took years of questions, testing, and advocacy to get answers.
Today, she loves walks, playing in the water, and Peppa Pig, and she has a smile that lights up a room.
She is currently receiving personalized treatment through the n-Lorem Foundation.
If you’d like to support patients like Anais and the research making treatment possible, you can learn more here: https://www.nlorem.org/support/
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