N-Acetyl Mannosamine as a Therapeutic Agent

N-Acetyl Mannosamine is a precursor for the synthesis of sugar molecules known as sialic acids which play an important role in specific biological processes such as cellular adhesion, cellular communication and signal transduction. Lack of sialic acids also play an important role in disease processes such as cancer, inflammation and immunity.

This invention relates to methods of administering N-Acetyl Mannosamine or its derivative (to produce sialic acid in patients who are deficient in the sugar molecule) to treat muscular atrophy including hereditary inclusion body myopathy (HIBM) and distal myopathy with rimmed vacuoles (Nonaka myopathy). Certain kidney conditions such as those arising from hyposialytion of kidney membranes may be treated by this method as well.

Potential Commercial Applications: Competitive Advantages:
  • Treatment of rare diseases such as HIBM and Nonaka myopathy.
  • Treatment of kidney conditions involving sialic acid deficiencies resulting in proteinuria and hematuria.
  • May be useful in treating other diseases involving sialic acid deficiencies.


Marjan Huizing (NHGRI)  ➽ more inventions...

Intellectual Property:
U.S. Pat: 8,410,063 issued 2013-04-02
U.S. Pat: 9,095,597 issued 2015-08-04
U.S. Pat: 10,335,426 issued 2019-07-02
US Application No. 14/754,304
US Application No. 13/791,576
US Application No. 15/702,529
US Application No. 16/417,488

Galeano B, et al. PMID 17549255

Collaboration Opportunity:

The National Human Genome Research Institute, Medical Genetics Branch, Cell Biology of Metabolic Disorders unit is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize N-acetylmannosamine as a therapeutic agent. Please contact Marjan Huizing at 301-402-2797or mhuizing@mail.nih.gov for more information.

Licensing Contact:
Anna Solowiej,
Email: solowieja@mail.nih.gov
Phone: 301-435-7791

OTT Reference No: E-217-2007-0
Updated: Sep 23, 2015