A Pilot Study of Bone Marrow Transplantation in a GALT‐Null Rat Model of Classic Galactosemia

ABSTRACT Classic galactosemia (CG) is a rare inborn error of metabolism with substantial unmet medical need. Early detection, often by population newborn screening, enables immediate and life‐long dietary restriction of galactose, which is the current standard of care. This treatment minimizes or pr...

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出版年:	JIMD Reports
主要な著者:	Shauna A. Rasmussen, Madelyn M. Seemiller, Ingrid Smith, Madeleine Wilson, Jennifer M. I. Daenzer, Keenan Wiggins, Judith L. Fridovich‐Keil
フォーマット:	論文
言語:	英語
出版事項:	Wiley 2025-07-01
主題:	bone marrow transplantation galactosemia GALT‐null metabolic efficacy rat model
オンライン･アクセス:	https://doi.org/10.1002/jmd2.70037