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Orphazyme A/S

http://www.orphazyme.com

Latest From Orphazyme A/S

Rare Diseases Rule, But Some Common Conditions Stand Out In Pending US Applications

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

Rare Diseases Drug Approval Standards

US FDA’s Genetic Metabolic Diseases Adcomm Debuts With Zevra’s Arimoclomol

The new panel’s inaugural 2 August meeting will take up a challenging drug application, currently in its second review cycle, for Niemann-Pick disease type C, an ultra-rare condition without approved therapeutics.

Advisory Committees Rare Diseases

BIO: Upbeat Zevra Advances Promising Rare Disease Portfolio

With the urea cycle disorder treatment Olpruva launched and the NPC therapy arimoclomol closing in on US approval, Zevra is one to watch in the rare disease space.

Business Strategies Rare Diseases

ADDF Leaders Discuss Future Strategies In Alzheimer’s Research And Crucial Role Of Diagnostic Markers

Medtech Insight talked to two leaders at the Alzheimer’s Drug Discovery Foundation about current therapies and the role of biomarkers and digital tools to build on recent advancements in the space and accelerate progress to identify the disease early and slow cognitive decline.

BIO Neurology
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Company Information

  • Industry
  • Pharmaceuticals
  • Biotechnology
    • Large Molecule
  • Other Names / Subsidiaries
    • Orphazyme US, Inc.
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