When a three-year-old from California flies to Manchester for a one-off experimental infusion, it is not just a family making a bet on science. It is a glimpse of a new kind of “passport medicine” in which access to gene therapy depends as much on geography, screening and logistics as on biology.
A rare disease that cannot wait
Hunter syndrome – or mucopolysaccharidosis type II (MPS II) – is the kind of diagnosis that collapses a family’s sense of time.
Children are usually born looking healthy. Somewhere between their second and fourth birthdays, parents start to see small things: frequent ear infections,
