What this study looked at
Delivering enzyme replacement therapy for CLN2 disease (a form of Batten disease) means infusing it directly into the brain's ventricles, which is more demanding than an ordinary drip. The Hamburg NCL Specialty Clinic — the first centre in the world to give this treatment — analysed more than 3,000 infusions in 48 patients over six years to develop best-practice safety guidelines.
Key takeaways
- Infusion into the ventricles every two weeks carries a higher infection risk than intravenous therapies, so careful protocol matters.
- Following standardised procedures kept device-related adverse-event and infection rates very low, at 0.27% and 0.33% respectively.
- The work produced the Hamburg Best Practice Guidelines for intraventricular enzyme replacement therapy, offering practical guidance for any centre setting up these infusions.
Why it matters
It makes a demanding treatment safer to deliver as more centres begin to offer it.
