Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience - Concepedia

Concepedia

Publication | Open Access

Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience

DOI Full Paper Access

23

Citations

32

References

2020

Year

Rita Fischetto, Valentina Palladino, Maria Margherita Mancardi, Thea Giacomini, Stefano Palladino, Alberto Gaeta, Maja Di Rocco, Lucia Zampini, Giuseppe Lassandro, Vito Favia,

Molecular Genetics & Genomic Medicine

Abstract

This treatment was safe and relatively well tolerated by all patients, with stabilization and/or slowing down of the neurological progression in three subjects.

References

	Year	Citations

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