Mucopolysaccharidosis type IIIB syndrome (also known as Sanfilippo type B syndrome) is a lysosomal storage disease resulting in progressive deterioration of cognitive acquisition after age 2-4 years. No treatment is available for the neurological manifestations of the disease. We sought to assess the safety and efficacy of a novel intracerebral gene therapy.

中文翻译：

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脑内基因治疗儿童黏多糖贮积症IIIB型综合征：一项未经控制的1/2期临床试验。

粘多糖贮积症IIIB型综合征（也称为Sanfilippo B型综合征）是一种溶酶体贮积病，会导致2-4岁后认知能力的逐渐恶化。没有针对该疾病的神经学表现的治疗方法。我们试图评估一种新型脑基因治疗的安全性和有效性。