Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience.
Rita FischettoValentina PalladinoMaria M MancardiThea GiacominiStefano PalladinoAlberto GaetaMaja Di RoccoLucia ZampiniGiuseppe LassandroVito FaviaMaria E TripaldiPietro StrisciuglioAlfonso RomanoMariasavina SeverinoAmelia MorronePaola GiordanoPublished in: Molecular genetics & genomic medicine (2020)
This treatment was safe and relatively well tolerated by all patients, with stabilization and/or slowing down of the neurological progression in three subjects.