"My grandson is devastated," he said, "but we've resigned ourselves to the fact that we've got to go".
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。
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