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GM1 gangliosidosis type 1
Review Articles from PubMed
Type 1 GM1 gangliosidosis is an ultra-rare, rapidly fatal lysosomal storage disorder, with life expectancy of <3 years of age. To date, only one prospective natural history study of limited size has been reported. Thus, there is a need for additional research to provide a better ...
Editorials from PubMed
Research Articles from PubMed
N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for G-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type ...