Case Report: Novel treatment approach for severe interstitial lung disease in type 3 Gaucher disease

Case Report: Novel treatment approach for severe interstitial lung disease in type 3 Gaucher disease

Gaucher Disease Type 3 (GD3) is a rare lysosomal storage disorder characterized by both visceral and neurological involvement. Pulmonary manifestations can significantly impact prognosis and quality of life. This case report highlights the challenges in managing severe pulmonary involvement in GD an...

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Bibliographic Details
Main Authors: Vincenza Gragnaniello, Silvia Carraro, Tiziana Zangardi, Chiara Cazzorla, Daniela Gueraldi, Alberto B. Burlina
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-06-01
Series:Frontiers in Pediatrics
Subjects:
Gaucher disease
glucocerebrosidase
newborn screening
interstitial lung disease
corticosteroid therapy
hydroxychloroquine
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2025.1604433/full
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