The Safety and Efficacy of Adalimumab for Pain Reduction in Individuals with Mucopolysaccharidoses: A Phase 1/2 Randomized, Double-Blind, Placebo-Controlled Trial

阿达木单抗用于减轻粘多糖症患者疼痛的安全性和有效性：1/2 期随机、双盲、安慰剂对照试验

• 批准号: 474611
• 负责人: Mitchell John J
• 金额: $ 21.39万
• 依托单位: Research Institute of the McGill University Health Centre
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2022
• 资助国家: 加拿大
• 起止时间: 2022-07-01 至 2025-07-01
• 项目状态: 未结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=474611&lang=en
• 关键词: Growth; Mucopolysaccharidoses; Pain; Quality Of Life; Rare Disease; Tnf-Alpha

Mucopolysaccharidoses (MPS) is a group of rare diseases associated with skeletal abnormalities, joint stiffness, short stature, pain, and, in some subtypes, cognitive disabilities. Although treatments have improved life expectancy and cognitive outcomes f

粘多糖病（MPS）是一组罕见疾病，与骨骼异常、关节僵硬、身材矮小、疼痛以及某些亚型的认知障碍有关。虽然治疗改善了预期寿命和认知结果