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Updated: Jul 7, 2025

Determination of Glucan Chain Length Distribution of Glycogen Using the Fluorophore-Assisted Carbohydrate Electrophoresis FACE Method
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在粘多糖症 (Sanfilippo综合征) 型IIIAIA多个领域的能力变化

Elsa G Shapiro1, Julie B Eisengart2, David Whiteman3

  • 1Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA; Shapiro Neuropsychology Consulting, LLC, Portland, OR, USA.

Molecular genetics and metabolism
|December 27, 2023
PubMed
概括

增长尺度值 (GSVs) 有效地追踪了IIIA粘多糖症 (Sanfilippo综合征A型) 的技能下降,特别是在认知方面. 这一新指标显示了MPS IIIA未来临床试验的前景,有助于检测功能变化.

关键词:
临床终点是临床终点.临床试验中的临床试验.增长规模的价值增长规模的价值.测量方法 测量方法粘多糖症 IIIAIA 粘多糖症神经认知能力下降的神经认知能力下降

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