Det er yderst vigtigt at behandle unge patienter i alderen < 5 år, der lider af en svær form af sygdommen, endda skønt patienter < 5 år ikke var inkluderet i det yderst betydningsfulde fase 3 studium.
A key issue is to treat young patients aged < 5 years suffering from a severe form of the disease, even though patients < 5 years were not included in the pivotal phase 3 study.