A Carlsbad-based nonprofit foundation is offering a glimmer of hope to the family of a 3-year-old girl diagnosed with a rare and terminal neurodegenerative disease. Harlow Graskewicz-Prado, who suffers from a condition known as leukodystrophy caused by a mutation in the TUBB-4A gene, is receiving experimental treatment through n-Lorem, a foundation dedicated to providing free personalized medicine for individuals with ultra-rare diseases.
The disease, so uncommon that it lacks an official name, affects an estimated 70 individuals worldwide. Harlow’s symptoms first appeared when she was unable to walk at two years old. Genetic testing, following an MRI, confirmed the diagnosis. Her mother, Daphyne Graskewicz-Prado, shared her distress upon receiving the news. “When you have a kid and you know something is wrong and you’re waiting for test results, you go on TikTok, YouTube,” she said. “I prayed to God, just don’t let it be leukodystrophy, because I know my baby was going to die.”
Determined to find a solution, the family relocated from southern Illinois to San Diego to access the life-saving treatment developed by n-Lorem. The foundation, founded by Dr. Stan Crooke, has pioneered the use of Antisense technology to treat rare genetic disorders like Harlow’s.
Dr. Crooke, an innovator in RNA-targeted therapies, established n-Lorem with the mission of applying cutting-edge medical technology to help individuals battling rare diseases. “These diseases destroy not just patients but families,” Dr. Crooke said. “I decided to start n-Lorem to try to bring the technology we had invented to treat millions now back down to treat a single human being—and to do that for free for life.”
While n-Lorem is providing the personalized medication free of charge, the costs associated with Harlow’s treatment are substantial. The family is expected to incur over $100,000 in expenses for the administration of the treatment at Rady’s Children’s Hospital, as insurance does not cover experimental therapies. Harlow’s father, a member of the military, along with her two siblings, has launched a fundraising campaign to cover these costs. Thanks to the support of friends, family, and the community, the family has raised enough funds through GoFundMe to cover two years of care.
The treatment is set to begin in April, but doctors have made it clear that Harlow will need lifelong therapies to manage her condition. “When you get the diagnosis, you don’t feel that. You get hit in the gut, punched in the gut,” Graskewicz-Prado said. “It’s like we have to find a path forward somewhere. n-Lorem is that hope.”
As the family continues their journey with Harlow’s treatment, they remain hopeful that the breakthrough therapy will improve her quality of life and provide a chance for survival.
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