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Research programme: lysosomal storage disease therapeutics - NanoMedSyn

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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: 20 Mar 2024

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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

  • 20 Mar 2024 Lysosomal storage disease therapeutics is still in preclinical trials for Glycogen-storage-disease-type-II and Lysosomal storage diseases in France (NanoMedSyn pipeline, March 2024)
  • 28 Apr 2022 No recent reports of development identified for preclinical development in Lysosomal storage diseases in France
  • 28 Sep 2020 No recent reports of development identified for preclinical development in Glycogen-storage-disease-type-II in France

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