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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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Principais autores: Renuka P. Limgala, Ozlem Goker-Alpan
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI AG 2020-03-01
coleção:Biomolecules
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Acesso em linha:https://www.mdpi.com/2218-273X/10/4/526
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