Young Girl Cleared for Gene Therapy Treatment
Six-year-old Lottie Milburn passed an essential antibody test required to receive the newly approved Fayuvi treatment.
Updated on Oct. 8, 2026 in Biotech
Six-year-old Springfield resident Lottie Milburn has passed the antibody screening necessary to begin treatment with Fayuvi. The FDA approved this gene therapy on September 17 as the first treatment for children suffering from Sanfilippo syndrome type A.
Why it matters
Sanfilippo syndrome is a rare condition that causes progressive, irreversible damage to the brain and nervous system. Fayuvi works by using a modified virus to deliver a functional gene copy, enabling cells to produce the enzyme required to break down heparan sulfate.
The treatment protocol requires corticosteroid use for at least eight weeks following the one-time intravenous infusion. Fayuvi is specifically indicated for children like the six-year-old patient who meet strict antibody screening criteria.
The players
Lottie Milburn
She is a six-year-old Springfield resident who was diagnosed with Sanfilippo syndrome in 2025.
Nikki Budzinski
She is a Congresswoman who plans to support legislation aimed at expanding newborn screenings for rare diseases.
St. Louis Children's Hospital
It is the medical facility where the Milburn family expects the intravenous infusion procedure will take place.
The details
The gene therapy aims to halt or slow the devastating progression of Sanfilippo syndrome, which leads to neurological decline. Lottie Milburn, who was diagnosed with the condition in April 2025, is now preparing for the infusion procedure at St. Louis Children's Hospital.
Timeline
April 2025: Lottie Milburn received her initial diagnosis.
September 17, 2026: The FDA approved Fayuvi for Sanfilippo syndrome type A.
October 5, 2026: Lottie Milburn passed the required antibody test.
The Big Picture
This development underscores the importance of the newborn screening for rare diseases legislative initiative, as early diagnosis remains essential for accessing emerging gene therapies.
The approval of this gene therapy provides a potential path to slow disease progression for children who pass rigorous eligibility screenings. Families must coordinate long-term monitoring and follow strict corticosteroid requirements after the procedure.
The takeaway
Advancements in gene therapy are creating new opportunities for children with rare genetic conditions to receive life-changing medical interventions. Families affected by similar diagnoses should consult with specialist centers to monitor eligibility for emerging FDA-approved protocols.
Further reading
Learn more about the latest developments in Biotech to understand the evolving landscape of rare disease treatments.
Source note: This article includes information reported by Illinois Times, the capital city's weekly source of news, politics, arts, entertainment, culture.
