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

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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Bibliographic Details
Main Authors: Shauna A. Rasmussen, Madelyn M. Seemiller, Ingrid Smith, Madeleine Wilson, Jennifer M. I. Daenzer, Keenan Wiggins, Judith L. Fridovich‐Keil
Format: Article
Language:English
Published: Wiley 2025-07-01
Series:JIMD Reports
Subjects:
bone marrow transplantation
galactosemia
GALT‐null
metabolic efficacy
rat model
Online Access:https://doi.org/10.1002/jmd2.70037
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