The European consortium for Pompe disease has updated its recommendations on enzyme replacement therapy (ERT) for this metabolic myopathy. The considerable hindsight now available on this treatment, its potential benefits and its real-life results were taken into account, as was the arrival of alternative (and/or new-generation) therapies.
Three topics in particular were addressed by the experts,
- when to start treatment (S for start),
- when and how to switch to an alternative treatment (S for switch),
- and above all, when to stop ERT (S for stop), and on what criteria.
There is no doubt that this consensus will help practitioners in the field to better manage ERT in everyday practice.
