14800-DvRappard

44 Chapter 2 are promising, but more information is needed regarding the safety and long-term outcomes of this therapy. HSCT does not seem to be beneficial for overtly symptomatic patients or patients with the aggressive late-infantile onset type. The results for asymptomatic juvenile and adult patients are more encouraging. In order to come to a valid conclusion, a systematic evaluation including a larger patient sample, and longer follow-up periods is necessary.