Article
Glucocorticoid Steroid and Alendronate Treatment Alleviates Dystrophic Phenotype with Enhanced Functional Glycosylation of α-Dystroglycan in Mouse Model of Limb-Girdle Muscular Dystrophy with FKRPP448L Mutation.
The American journal of pathology - 1 Jun 2016
Wu Bo, Shah Sapana N, Lu Peijuan, Richardson Stephanie M, Bollinger Lauren E, Blaeser Anthony, Madden Kyle L, Sun Yubo, Luckie Taylor M, Cox Michael D, Sparks Susan, Harper Amy D, Lu Qi Long
Abstract excerpt
Fukutin-related protein-muscular dystrophy is characterized by defects in glycosylation of α-dystroglycan with variable clinical phenotypes, most commonly as limb-girdle muscular dystrophy 2I. There is no effective therapy available. Glucocorticoid steroids have become the standard treatment for Duchenne and other muscular dystrophies with serious adverse effects, including excessive weight gain, immune...
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