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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"So we want to just take advantage of this to set up both vendors for future success on a lunar landing," he said. "This is the proper way to do it, if it works out from a timing perspective, to be able to rendezvous and dock with both. ... This, again, is the right way to proceed in order to have a high confidence opportunity in '28 to land."