A local Southwest RivCo family is celebrating a breakthrough in their fight to save their 3-year-old daughter’s life after the community rallied behind them to fund a rare gene therapy treatment.
Harlow, the daughter of Daphne and Luis, was diagnosed in July 2023 with TUBB4A leukodystrophy, a rare and fatal neurodegenerative disease. Without intervention, the condition would progressively take away her ability to walk, eat, speak and, ultimately, her life.
A gene therapy that offers hope
The n-Lorem Foundation is a nonprofit providing individualized treatments free of charge for patients with ultra-rare diseases. The U.S. Food and Drug Administration approved Harlow’s clinical trial and Investigational New Drug application, paving the way for treatment at Rady Children’s Hospital in San Diego.
While the therapy itself is provided at no cost, the administration of the trial carried unexpected expenses not fully covered by insurance. The Johnson family faced more than $120,000 in costs and turned to the community for help.
A community response
The family launched a GoFundMe campaign in February of this year and quickly saw an outpouring of support. As of this article more than 3.3K donations have poured in, and the initial goal was met in less than four days.
“We put out a plea for help and you all responded with a battle cry for our daughter,” Daphne and Luis wrote in an update earlier this year. “You became part of Harlow’s fight, part of her miracle.”
In September, Daphne confirmed that Harlow’s first treatment was officially scheduled for October, making her only the second child in the world to receive therapy for TUBB4A leukodystrophy.
The family expressed deep gratitude to their community, military supporters, Peloton parent groups and others who amplified Harlow’s story. “Through coverage of Harlow’s journey, I know others won’t have that experience of Googling this disease and seeing no hope,” Daphne told Valley News. “They will know there is hope that exists.”
Ongoing journey
Although the initial fundraising goal has been reached, the GoFundMe remains open to help with ongoing medical costs and to support the family as they continue Harlow’s care.
Supporters have left words of encouragement across social media and the fundraiser page. “I hope you get all of the donations you need for sweet baby Harlow,” wrote donor Melissa Cohen. Another supporter, Leigh Crow, shared Harlow’s story with multiple online communities: “I saw your post in the Peloton Moms group and have been spreading the word far and wide.”
For now, the family says they are focused on treatment and transparency, pledging to keep the community updated on Harlow’s progress.
“We’ll share the highs and the lows, the progress and the challenges, so that you can walk this road with us,” Daphne wrote in a public update. “From the bottom of our hearts, thank you for being part of Harlow’s story.”
How to help
The fundraiser, “Save Harlow – Help Her Access Life-Saving Gene Therapy” is still accepting donations at GoFundMe.com.
