Yukiko Kimura

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OBJECTIVE Gaucher disease (GD) is a lysosomal storage disease characterized by a deficiency of glucocerebrosidase. Although enzyme-replacement and substrate-reduction therapies are available, their efficacies in treating the neurological manifestations of GD are negligible. Pharmacological chaperone therapy is hypothesized to offer a new strategy for(More)
OBJECTIVE There is wide variation in therapeutic approaches to systemic juvenile idiopathic arthritis (JIA) among North American rheumatologists. Understanding the comparative effectiveness of the diverse therapeutic options available for treatment of systemic JIA can result in better health outcomes. The Childhood Arthritis and Rheumatology Research(More)
OBJECTIVE To evaluate the interleukin-6 receptor inhibitor tocilizumab for the treatment of patients with polyarticular-course juvenile idiopathic arthritis (pcJIA). METHODS This three-part, randomised, placebo-controlled, double-blind withdrawal study (NCT00988221) included patients who had active pcJIA for ≥6 months and inadequate responses to(More)
The proven effectiveness of biologics and other immunomodulatory products in inflammatory rheumatic diseases has resulted in their widespread use as well as reports of potential short- and long-term complications such as infection and malignancy. These complications are especially worrisome in children who often have serial exposures to multiple(More)
OBJECTIVE To determine the methods of anesthesia currently being used by pediatric rheumatologists when performing intra-articular corticosteroid injections (IACI). STUDY DESIGN A questionnaire was emailed to all members of the Childhood Arthritis & Rheumatology Research Alliance, a pediatric rheumatology research network in North America. The(More)
UNLABELLED BACKGROUND The management of background corticosteroid therapy in rheumatology clinical trials poses a major challenge. We describe the consensus methodology used to design an algorithm to standardize changes in corticosteroid dosing during the Randomized Placebo Phase Study of Rilonacept in Systemic Juvenile Idiopathic Arthritis Trial(More)
BACKGROUND Intra-articular corticosteroid injections (IACI) are routinely used by pediatric rheumatologists in the treatment of chronic arthritis. Frequently, topical anesthetics are used to control procedural pain, but their relative efficacy has not been reported. In this study, we evaluated the level of pain associated with different anesthetic methods,(More)
Purpose Currently, there is significant variability in the therapeutic approaches to new onset systemic Juvenile Idiopathic Arthritis (sJIA), as evidenced by surveys using case presentations administered to Childhood Arthritis and Rheumatology Research Alliance (CARRA) members. Understanding the comparative effectiveness of these diverse therapeutic(More)