Discussion
DISEASES, BLIND
Given the lack of global symptomatic or disease-modifying therapies for NPC and other lysosomal diseases, there is an urgent need for effective and well-tolerated drug treatments. This multi-center, multinational randomized double- blind placebo-controlled crossover phase III trial was designed through a collaboration between national regulatory agencies, leading clinical experts, patient organizations, and the industry sponsor. This design addresses the unique ethical and practical challenges of conducting clinical trials for these orphan, heterogeneous patient populations in order to better capture N-acetyl-L-leucine’s therapeutic effect and thus expedite the development and availability of this promising drug candidate [