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Updated: Sep 15, 2025

Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index
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美国 propionic acidemia 的临床负担:按年龄层分类的基于索赔的研究

Geetanjoli Banerjee1, Sue Perera1, Fan Mu2

  • 1Moderna Therapeutics, Inc., 325 Binney St, Cambridge, Massachusetts, 02142, USA.

Orphanet journal of rare diseases
|July 15, 2025
PubMed
概括

患有酸血症 (PA) 的患者在所有年龄段都会出现重大健康问题和并发症. 这项研究强调了与对照组相比,它们的疾病负担增加,强调了需要更好的治疗方法的必要性.

关键词:
行政索赔数据 管理索赔数据年龄分层分类 年龄分层分类临床结果 临床结果代谢性失补偿事件发生.普罗皮昂酸血症 普罗皮昂酸血症普罗皮尼尔辅酶A类碳氧化酶

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