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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。
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Jacqui Gabb, Professor of Sociology and Intimacy at The Open University, assessed this in her Enduring Love project, published in the journal Sociology in 2015.
Sue Peacock was diagnosed with gallstones and told she would have to have her gallbladder removed
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