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Method of treatment of Shwachman-diamond syndrome
Method of treatment of Shwachman-diamond syndrome
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机译:SHUCHMAN-钻石综合征的治疗方法
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摘要
The invention relates to compounds for the treatment of ribosomopathies. In particular, it refers to compounds for the treatment of Shwachman-Diamond Syndrome (SDS) and other ribosomopathies, such as Diamond-Blackfan Anemia (DBA), X-linked dyskeratosiscongenita (DKC) and Treacher Collins syndrome (TCS).
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