Problems associated with New Therapy for Patients with Rare intractable disease child and their Families

粘多糖病是一种先天性、遗传性的溶酶体病，目前尚无特效的治疗方法。2007年，一种使用Elaprase的酶置换疗法被引入日本，Elaprase是一种期待已久的粘多糖病治疗剂(基因重组)。本研究旨在探讨黏多糖症治疗给患者及其家属带来的利益与负担，并为接受治疗的焦虑症患者提供持续的支持。受益组的写作可进一步分为身体状况、心理社会学和发展3类，负担组的写作可进一步分为治疗方法、护理、医疗效果和医疗设施与系统4类。

Mucopolysaccharidosis is one of lysosomal diseases that are congenital and hereditary inborn errors of metabolism , and currently we ’ve found no specific treatment for full recovery from mucopolysaccharidosis . In 2007 , an enzymereplacement therapy with Elaprase , a long-awaited therapeutic agent (genetical recombination) for mucopolysaccharidosis , was introduced to Japan . This study aims to research the benefit and burden the patients and their families are suffering due to the therapy for mucopolysaccharidosis , and to define an approach to consistently support the patients who are under the therapy with anxiety . The writingorganized in the benefit group could be further divided into 3 categories : bodycondition , psychology and sociology , and development , whileas the writing in the burden group could be further divided into 4 categories of therapy approach , nursing care , medical effect , and medical facility and system.