Dr Ruqaiah Altassan describes the successful heart transplantation in a child with PGM1-CDG.

Successful heart transplantation in an infant with phosphoglucomutase 1 deficiency (PGM1-CDG)

Ruqaiah Altassan, et al

https://doi.org/10.1002/jmd2.12350

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AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase I deficiency (PGM1-CDG).

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