New #treatment for #rare metabolic disease

Scott Garman, University of Massachusetts Amherst, Fabry, rare disease, orphan drugs, National Institute of Health, World Orphan Drug Congress, pharma, biotech An investigation team led by biochemist Scott Garman at the University of Massachusetts Amherst discovered a genetic interaction that can be a treatment for Fabry disease.

Garman, Associate Professor of Biochemistry and Molecular Biology, explains: “The interactions we looked at are exactly the things occurring in the clinical trial right now. The same concept is now being applied to other protein-folding diseases such as Parkinson’s and Alzheimer’s diseases.”

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The studies, supported by the National Institute of Health, are definitely advancing the understanding of orphan drugs for the rare childhood metabolic disorder.

It is extremely important to support and discuss new strategies to improve the orphan drugs market and help treatments for rare diseases. Experts and executives from the leading pharma and biotech companies will be at the World Orphan Drug Congress USA 2012.

Learn now how to complete pre-clinical and clinical trials with a very widespread patient group, find out the most innovative orphan drug technologies and strategic solutions for biomakers, biosimilars and more.

Register now and don't miss the opportunity to be present in the orphan drug space interactions with business and scientific industry leaders from North America and beyond.