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Research programme: pharmacological chaperones, enzyme replacements and co-formulations - Amicus Therapeutics

Drug Profile

Research programme: pharmacological chaperones, enzyme replacements and co-formulations - Amicus Therapeutics

Alternative Names: AT 2221; AT3375; ATB 101/Migalastat; Chaperone monotherapy - Amicus Therapeutics; Chaperone-Enzyme Replacement Therapy - Amicus Therapeutics; Chaperone-ERT combinations - Amicus Therapeutics; Duvoglustate/rhGAA; rhGAA/AT-2220; rhGAA/duvoglustat

Latest Information Update: 28 Feb 2018

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At a glance

  • Originator Amicus Therapeutics
  • Class Alkaloids; Alpha-glucosidases; Glycoside hydrolases; Imino pyranoses; Pharmacological chaperones; Small molecules
  • Mechanism of Action Alpha glucosidase replacements; Glucosylceramidase replacements; Iduronidase replacements; Protein folding stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity Yes

Highest Development Phases

  • Research Fabry's disease
  • No development reported Alzheimer's disease; Gaucher's disease; Glycogen storage disease type II; Mucopolysaccharidosis I; Parkinson's disease

Most Recent Events

  • 28 Feb 2018 No recent reports of development identified for preclinical development in Glycogen-storage-disease-type-II(Combination therapy) in USA (SC)
  • 15 Jan 2018 Early research in Fabry's disease (Combination therapy) in USA (IV)
  • 08 Jan 2018 Preclinical trials in Fabry's disease (Combination therapy) in USA (IV)
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