In this issue:
M021 in Gaa-knock-out mice
Mouse model of a GYS1-inhibitor
Anti-hTfR-GAA in knock-in mice
GAA enzyme & glycogen levels in healthy individuals
ERT for LOPD diagnosed via newborn screening
Lower-limb antigravity movement as a predictor of functional outcome in IOPD
Newborn screening for lysosomal disorders in Indiana
Managing LOPD identified via newborn screening
Benefits & harms of uncertain prognoses in newborn screening
Treatment-related hyperglycaemia
MCIDs for 6-minute walking distances in LOPD
Ptosis measurements in IOPD
Long-term outcomes of IOPD patients treated with ERT
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