加拿大研究表明,药物4-PBA可能有助于治疗稀有遗传疾病、桑道夫和Tay-Sachs。 Canadian study shows drug 4-PBA may help treat rare genetic disorders, Sandhoff and Tay-Sachs.

加拿大研究人员发现,由林业发展局批准的一种现有药物,即4-苯丁酸(4-PBA),可能会改善患有罕见遗传病Sandhoff和Tay-Sachs的患者的生活质量。 Canadian researchers found that an existing FDA-approved drug, 4-Phenylbutyric Acid (4-PBA), may improve life quality for patients with rare genetic disorders Sandhoff and Tay-Sachs. 这种用小鼠测试的药物表明,运动功能、寿命延长以及健康神经元的增加都有改善。 The drug, tested in mice, showed improvements in motor function, extended lifespan, and increased healthy neurons. 这一发现还有助于研究其他神经退化性疾病,如阿尔茨海默氏病和ALS。 This discovery could also inform research on other neurodegenerative diseases like Alzheimer's and ALS.