宝马基金会正在努力寻找治愈方法,但谁拥有这种疾病?
Luuk Kerckhaert1, Pieter Glijnis1, Annet N Linders1
1PLN Foundation, Wieringerwerf, The Netherlands.
概括
该PLN基金会帮助个人与phospholamban (PLN) 基因突变心肌病. 它提出了知识产权共同所有权和患者领导等解决方案,以克服研究障碍并加速罕见疾病治疗.
科学领域:
- 生物医学研究生物医学研究
- 罕见疾病治疗方法 罕见疾病治疗方法
- 患者倡导 患者倡导
背景情况:
- 该PLN基金会支持1700个人与索兰班 (PLN) 基因突变诱导心肌病.
- 与学术机构和生物技术公司的合作对于开发这种罕见遗传疾病的治疗方法至关重要.
研究的目的:
- 在研究伙伴关系中识别和解决患者基金会面临的挑战.
- 为加强合作和加快治疗罕见疾病的发展提出战略.
主要方法:
- 分析学术界-工业界-患者合作中的障碍.
- 在研究机构和技术转让办公室 (TTO) 中倡导政策和程序变化.
主要成果:
- 确定了机构利益,出版或灭亡文化,专利纠纷和官僚过程是主要障碍.
- 建议的解决方案包括知识产权共同所有权,患者领导,简化协议和修订的科学评估指标.
结论:
- 克服制度和官僚障碍对于有效的罕见病研究至关重要.
- 实施拟议的战略可以促进共同创造,加速药物开发,并改善治疗的可获得性.
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