"Our biggest success is keeping on the traditions we have had over the last 75 years, and seeing them passed down from generation to generation. There have been difficult times, but we have kept going," he says.
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Read more global business storiesThe shadowy world of abandoned oil tankers
Vosoritide的成功,再次向外界证明了罕见病赛道的巨大潜力:ACH看似是罕见病,但明确的致病机制、典型的临床表现,叠加数万级的患者基数和迫切、长期的治疗需求,使其成为了一个极具开发价值的 “不罕见” 罕见病市场。