Improvements in liver and lipid outcomes continue in children and adults with chronic acid sphingomyelinase deficiency treated for 2 to 6.5 years with olipudase alfa in long-term clinical trials
Robin Lachmann, Jaya Ganesh, Roberto Giugliani, Nathalie Guffon, Eugen Mengel, Maurizio Scarpa, Melissa P. Wasserstein, Mario Aguiar, Nicole Armstrong, B. Thurberg, Monica Kumar
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