AAVANTGEDE 生物治疗 Usher综合症1B型失明 获得FDA 孤儿药物的地位。 AAVantgarde Bio's treatment for Usher Syndrome Type 1B blindness gets FDA orphan drug status.

意大利一家生物技术公司Aavantgarde Bio接受了FDA为AAVB-081指定的孤儿药物,这是对1B型Usher综合症(USH1B)视网膜炎色素的治疗。 AAVantgarde Bio, an Italian biotech firm, received FDA Orphan Drug Designation for AAVB-081, a treatment for Usher Syndrome Type 1B (USH1B) retinitis pigmentosa. 这一指定提供了税收抵免和市场排他性等奖励,旨在解决与USH1B有关的逐渐视力丧失和最终失明得不到治疗的问题,影响到约20,000美国和欧洲。 This designation, which offers incentives like tax credits and market exclusivity, aims to address the lack of treatment for the progressive vision loss and eventual blindness associated with USH1B, affecting around 20,000 U.S. and E.U. 患者。 patients.