MARKER FOR ACID SPHINGOMYELINASE DISORDERS AND USES THEREOF

• Main Authors: CHUANG WEI-LIEN, ZHANG X KATE, GERALD F COX
• Format: Patent
• Language: English; Japanese
• Published: 20.05.2021
• Subjects: HUMAN NECESSITIES; HYGIENE; INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIRCHEMICAL OR PHYSICAL PROPERTIES; MEASURING; MEDICAL OR VETERINARY SCIENCE; PHYSICS; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; TESTING

To provide a method of treating an acid sphingomyelinase (ASM) disorder.SOLUTION: A method comprises: (a) administering to a subject a first dose of a therapeutic agent for treating an ASM disorder having a first concentration; and (b) administering to the subject a second dose of the therapeutic agent having a second concentration equal to or greater than the first concentration if it is determined that the subject has a level of lyso-sphingomyelin (lyso-SPM) that is less than or equal to a reference level after administration of the first dose.SELECTED DRAWING: None 【課題】酸性スフィンゴミエリナーゼ（ＡＳＭ）障害を治療する方法を提供する。【解決手段】（ａ）対象に、第１の濃度を有する、ＡＳＭ障害を処置するための第１の用量の治療薬を投与すること；および（ｂ）対象が第１の用量の投与後、参照レベル以下のｌｙｓｏ−スフィンゴミエリン（ｌｙｓｏ−ＳＰＭ）のレベルを有することが決定された場合、対象に、第１の濃度以上の第２の濃度を有する第２の用量の治療薬を投与することを含む方法とする。【選択図】なし