Inventors at the National Human Genome Research Institute (NHGRI) discovered that N-acetylmannosamine (ManNAc) can be used for therapeutic purposes. ManNAc therapy given orally shows long-term safety and biochemical efficacy. ManNAc is a small uncharged physiological molecule that crosses membranes

NIH has 1,200 scientists in the Intramural Research Program who research and develop new drugs, vaccines, methods, and other products to better public health. These scientists become inventors when their research results in a licensable material or product. The NIH Record sat down with Dr. Niraj

Cobalamin C deficiency (cblC), caused by mutations in the MMACHC gene, is the most common inborn error of intracellular vitamin B12 metabolism. Inventors at the National Human Genome Research Institute (NHGRI) have developed a novel combination of hydroxo- and methylcobalamin, which has shown