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Boy, 8, Receives World's First Treatment for Rare Disease
6 Dec
Summary
- An 8-year-old boy is the first globally to receive treatment for a rare, unnamed disease.
- His mother tirelessly worked for years to develop and fund the experimental gene therapy.
- The groundbreaking FDA-approved treatment offers hope for lasting positive effects.
An 8-year-old boy named Maxwell has made medical history as the first person globally to receive treatment for a rare neurodevelopmental condition, SLC6A1. This disease, which can cause epilepsy, speech, and movement disorders, had previously gone unnamed.
Maxwell's mother, Amber Freed, spearheaded the effort to find a cure after his diagnosis in 2018. Despite initial discouragement, she committed years to fundraising and working with scientists to develop an experimental gene therapy. The COVID-19 pandemic caused significant delays, but perseverance led to the treatment's FDA approval on June 25, 2025.
In September 2025, Maxwell received the two-hour treatment, a pivotal moment celebrated by his family and medical team. While doctors caution it's too early to declare a cure, they are optimistic about lasting positive effects. Maxwell is now undergoing rehabilitation, with his progress impressing his doctors and offering hope for his future.
