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Substrate reduction therapy with miglustat in chronic GM2 gangliosidosis type Sandhoff: results of a 3-year follow-up.
Gabriella Silvestri*, Marcella Masciullo, Massimo Santoro, Pietro Tonali, Jeremy Guitton, Anna Modoni
*Corresponding author
Research output: Contribution to journal › Article
20
Citations
(Scopus)