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#AlphaMannosidosis
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Genetics in Medicine Open
Genetics in Medicine Open
@gimopenjournal.bsky.social
5 months ago
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#DelayedDiagnosis of alpha-mannosidosis is common especially in mild cases, highlighting the need for early diagnosis to facilitate timely treatment bit.ly/4oTXxpC #GIMO #AlphaMannosidosis #LysosomalStorageDisorder #DiseaseSeverity #RareDisease #EnzymeReplacementTherapy

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Journal of Inherited Metabolic Disease
Journal of Inherited Metabolic Disease
@jimd-editors.bsky.social
9 months ago
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🧬 New data on HSCT in alpha-mannosidosis just dropped!

21 children. 11 countries. Safer transplants, fewer infections, better hearing — and promising neurodevelopmental trends with early intervention.

📄 doi.org/10.1002/jimd...

#RareDisease #AlphaMannosidosis #HSCT #IMDs #PedsResearch #JIMD

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