The role of surfactant and distal lung dysfunction in the pathology of lysosomal storage diseases

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2021

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Paget, T.L.
Parkinson Lawrence, E.J.
Orgeig, S.

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Current Opinion in Physiology, 2021; 23(article no. 100467):1-8

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Respiratory dysfunction in lysosomal storage diseases (LSD) is primarily attributed to pulmonary obstruction due to the deposition of storage material in the upper and lower airways. In addition, skeletal deformities may contribute to lung disease. Recent cases of neonates presenting with respiratory distress suggest that interstitial lung disease may mask the symptoms of LSD. To date, very little attention has been directed at potential distal lung pathology involving the parenchyma, the pulmonary surfactant system and the small airways. In this review we summarise the literature on respiratory dysfunction in LSD and suggest that respiratory pathology has an independent aetiology that is not primarily consequent to neurological dysfunction.

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Copyright 2021

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