Score: 7ProsConsAttractive, retro design
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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。
"We don't know where they go, but they disappear for about 10 days, and then they'll come back again. And without the CCTV, we never knew that."