Substrate reduction therapy with miglustat in chronic GM2 gangliosidosis type Sandhoff: results of a 3-year follow-up

✍ Scribed by Masciullo, Marcella; Santoro, Massimo; Modoni, Anna; Ricci, Enzo; Guitton, Jerome; Tonali, Pietro; Silvestri, Gabriella

• Book ID: 111923033
• Publisher: Springer
• Year: 2010
• Tongue: English
• Weight: 427 KB
• Volume: 33
• Category: Article
• ISSN: 0141-8955
• DOI: 10.1007/s10545-010-9186-3