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.
Zdroj: In Genetics in Medicine Open 2024 2
Databáze: ScienceDirect