Research programme: lysosomal storage disease therapeutics - NanoMedSyn

Drug Profile

Research programme: lysosomal storage disease therapeutics - NanoMedSyn

Alternative Names: recombinant acid alpha-glucosidase conjugated with AMFA; Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues

Latest Information Update: 05 Apr 2018

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At a glance

  • Originator NanoMedSyn
  • Class Enzymes
  • Mechanism of Action Alpha glucosidase replacements; Enzyme replacements
  • Orphan Drug Status

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

    Yes - Glycogen storage disease type II
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Glycogen storage disease type II; Lysosomal-storage diseases

Most Recent Events

  • 26 Mar 2018 NanoMedSyn and Shire enter a research collaboration to develop lysosomal storage disease therapeutics
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