Substrate reduction therapy in Sandhoff disease: Evidence for improvement in nervous function in patients treated with miglustat

Eitemau Tebyg

• Miglustat - A viewpoint
  gan: Platt, F, et al.
  Cyhoeddwyd: (2003)
• Substrate reduction therapy reduces brain ganglioside GM2 in neonatal Sandhoff disease mice
  gan: Baek, R, et al.
  Cyhoeddwyd: (2005)
• Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease type C.
  gan: Lachmann, R, et al.
  Cyhoeddwyd: (2004)
• Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.
  gan: Elstein, D, et al.
  Cyhoeddwyd: (2004)
• Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease.
  gan: Andersson, U, et al.
  Cyhoeddwyd: (2004)