The efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: a systematic review.
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DOI: 10.1111/ene.15871
Abstract: BACKGROUND Since the results of previous studies regarding the safety and efficacy of miglustat in GM2 gangliosidosis (GM2g) were inconsistent, we aimed to assess the miglustat therapy in GM2g patients. METHODS This study followed the… read more here.
Keywords: safety; gm2 gangliosidosis; efficacy; treatment ... See more keywords
Ursodeoxycholic Acid Binds PERK and Ameliorates Neurite Atrophy in a Cellular Model of GM2 Gangliosidosis
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DOI: 10.3390/ijms24087209
Abstract: The Unfolded protein response (UPR), triggered by stress in the endoplasmic reticulum (ER), is a key driver of neurodegenerative diseases. GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff disease, is caused by an accumulation of GM2,… read more here.
Keywords: gm2 gangliosidosis; cellular model; gangliosidosis; ursodeoxycholic acid ... See more keywords