METHODS: We conducted an open-label, non-randomized, multi-center study into the effects of bezafibrate on five patients with VLCADD. Bezafibrate was administered for 4 years, and we analyzed the number of myopathic attacks requiring hospitalization and treatment infusions.
RESULTS: The number of myopathic attacks requiring infusions of 24 h or longer significantly decreased during the study period. The patients\' ability to conduct everyday activities was also improved by the treatment.
CONCLUSIONS: Our findings show the potential long-term efficacy of bezafibrate in preventing myopathic attacks for patients with VLCADD.