Reply to “Levacetylleucine a game changer for Niemann-Pick disease type-C”

This publication has 6 references indexed in Scilit:

• Long-term efficacy of intrathecal cyclodextrin in patients with Niemann-Pick disease type C
  Brain and Development, 2024
• Trial of N -Acetyl- l -Leucine in Niemann–Pick Disease Type C
  New England Journal of Medicine, 2024
• Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann–Pick Disease Type C Model Mice
  International Journal of Molecular Sciences, 2021
• Acetyl-leucine slows disease progression in lysosomal storage disorders
  Brain Communications, 2020
• Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
  PLOS ONE, 2009
• Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1 ^−/− mouse
  Proceedings of the National Academy of Sciences, 2009