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Fabry (stored globotriaosylceramide) using targeted enzyme replacement therapy with alpha-galactosidase A (e.g., Algalsidase alpha (Replagal marketed by Shire), Algalsidase beta (Fabrazyme marketed by Genzyme), or with substrate inhibition of glucosylceramide synthase with imino sugars (e.g., miglustat (Zavesca marketed by Actelion) = N-butyl-deoxynojirimycin (NB-DNJ), N-butyl-galactosyl-deoxynojirimycin (NB-DGJ), N-(5-adamantane-1-yl-methoxypentyl) (AMP-DNJ), AMP-DNM or MZ-21 and MZ-31 from Macrozyme), or with glucosylceramide analogs (e.g., Genz 112638: d-threo-ethylendioxyphenyl-2-palmitoylamino-3-pyrrilidino-propanol), or with chaperone therapy (AT1001, migalastat hydrochloride (to be marketed as Amigal by Amicus Therpeutics with chemical names 3,4,5-piperidinetriol, 2-(hydroxymethyl), hydrochloride, (2R,3S,4R,5S)-, (+)-(2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride, 1,5-dideoxy-1,5-imino-D-galactitol hydrochloride)

Tethering complexes in the endocytic pathway: CORVET and HOPS

[DOI] [PMC free article] [PubMed] [Google Scholar] 8.Leclerc D., Sibani S., Rozen R

By the three different assessment points, this indicates that none of the dietary variables were significantly changed over the course of the 8 weeks for either group, and that neither group differed from the other (Table 3)