ADO-RESISTANT CYSTEAMINE ANALOGS AND USES THEREOF

• Main Authors: LORNA DUFFY, ZANKEL TODD C, BENOIT GOURDET, TIMOTHY PHILLIPS, JOHN UNITT
• Format: Patent
• Language: English; Japanese
• Published: 30.09.2021
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS

To provide methods and substances for treating diseases for which treatment with cysteamine is indicated.SOLUTION: The present disclosure is directed to ADO-resistant cysteamine analogs for use in methods for treating diseases for which treatment with cysteamine is indicated, in particular, cystinosis, fatty liver disease, fibrosis, a thrombotic disease, an MECP-2 related disorder, an inherited mitochondrial disease, a neurological disease or disorder, inflammation and cancer. The present disclosure relates to methods and substances for treating diseases for which treatment with cysteamine is indicated. In particular, the present disclosure provides treatment methods including administering compounds disclosed herein to a patient.SELECTED DRAWING: Figure 1 【課題】 システアミンを用いた治療が適応である疾患を処置するための物質および方法を提供すること【解決手段】 本発明は、システアミンが適応である疾患、特に、シスチン蓄積症、脂肪肝疾患、線維症、血栓性疾患、ＭＥＣＰ−２関連障害、遺伝性ミトコンドリア病、神経疾患または障害、炎症およびがんを処置する方法における使用のためのＡＤＯ耐性システアミン類似体を対象とする。本開示は、システアミンを用いた治療が適応である疾患を処置するための物質および方法に関する。特に、本開示は、本明細書で開示されている化合物の患者への投与を含む治療方法を提供する。【選択図】図１