The impact of sphingomyelin on the pathophysiology and treatment response to olipudase alfa in acid sphingomyelinase deficiency
Autor: | Kumar, Monica, Aguiar, Mario, Jessel, Andreas, Thurberg, Beth L., Underhill, Lisa, Wong, Holly, George, Kelly, Davidson, Vanessa, Schuchman, Edward H. |
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Zdroj: | In Genetics in Medicine Open 2024 2 |
Databáze: | ScienceDirect |
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