USAResearch and clinical trials
Experimental gene therapy given into the brain fluid in Hurler syndrome: one child followed for 5 years
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In the USA, a 20-month-old boy with Hurler syndrome received an experimental gene therapy. It was given once into the brain fluid through a space at the back of the neck. The child was followed for more than 5 years, and side effects remained mild. He did not have a stem cell transplant, and weekly enzyme therapy continues. His development is behind that of children his age but better than the course without treatment.
What does it mean for patients and families?
This result comes from a single patient and does not give definitive information. At present, the treatment is available only within research.
Source
Scientific paper: First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT. Molecular Therapy, 2026. DOI: 10.1016/j.ymthe.2026.04.016 · PubMed 41966056
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Mucopolysaccharidosis type I (MPS I; Hurler/Scheie)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.