Development of a new drug for the treatment of Autism caused by Fragile X Syndrome

开发治疗脆性X综合征引起的自闭症新药

• 批准号: 102422
• 金额: $ 123.04万
• 依托单位: SENTINEL ONCOLOGY LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2015
• 资助国家: 英国
• 起止时间: 2015 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=102422
• 关键词: Development; new; drug; treatment; Autism

Fragile X Syndrome is the single largest cause of genetically inherited disability amongst diseasescategorised within the autism spectrum disorders.This proposal will aim to develop a new treatment for Fragile X syndrome, called SOL101, which willtreat the underlying cause of the disease rather than the symptoms. SOL101 has already demonstratedproof of concept in pre-clinical disease models and the proposal will develop it to the start of clinicaltrials.As well as the healthcare benefits to the patiens, in terms of alleviation of symptoms, both neurologicaland physical, the cost savings in terms of reduced needs for ongoing care and education assistance willbe considerable.Finance summary table

脆性X综合征是自闭症谱系障碍疾病中导致遗传残疾的最大原因。这项提案旨在开发一种治疗脆性X综合征的新方法，名为SOL101，它将治疗疾病的根本原因，而不是症状。SOL101已经在临床前疾病模型中证明了概念的正确性，该提议将把它发展到临床试验的开始。除了在缓解症状方面对患者的保健好处，在神经和身体方面，减少对持续护理和教育援助的需求方面的成本节约将是可观的。财务汇总表