Effect of Substrate Reduction Therapy in Comparison to Enzyme Replacement Therapy on Immune Aspects and Bone Involvement in Gaucher Disease

Gaucher disease (GD) is caused by mutations in the <i>GBA</i> gene, leading to deficient activity of the lysosomal enzyme glucocerebrosidase. Among all the symptoms across various organ systems, bone disease is a major concern as it causes high morbidity and reduces quality of life. Enzyme replaceme...

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Hauptverfasser:	Renuka P. Limgala, Ozlem Goker-Alpan
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	MDPI AG 2020-03-01
Schriftenreihe:	Biomolecules
Schlagworte:	Gaucher disease bone involvement enzyme replacement therapy substrate reduction therapy Osteoimmunology RANK/RANKL
Online-Zugang:	https://www.mdpi.com/2218-273X/10/4/526
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