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Wednesday, January 20, 2010

FDA Launches Orphan Drug Workshop at KGI : TMAU qualifies as an orphan disease

This February 25-26th sees the first 'Orphan drug workshop' at the Keck Graduate Institute in California. When they say 'orphan' they mean 'rare', and it is likely that trimethylaminuria qualifies under this grouping (though in truth it seems likely to be not rare. At least, when you include 'mild' cases. Odor is something that cannot be a hidden problem, which is probably the case with other 'mild' genetic disorders). It's very unlikely any company/researcher will be proposing a TMAU drug or treatment, although it does seem there could be potential treatments.

Examples of possible trimethylaminuria treatments :
Ataluren for genetic TMAU cases that are caused by nonsense mutations
A probiotic either naturally rich or genetically engineered to contain FMO3
TMA-dipsticks for urine testing monitoring
Stem cell therapy
TMAU dog (such as a guide dog)
etc

The U.S. Food and Drug Administration (FDA) has announced the launch of an orphan drug workshop series-an opportunity for biotechnology companies, pharmaceutical firms, academics and other interested parties to spend two days creating applications for orphan status designation, under the guidance of staff from FDA's Office of Orphan Products Development (OOPD). The first workshop is planned for Feb. 25-26, 2010, hosted by the Center for Rare Disease Therapies at Keck Graduate Institute (KGI) in Claremont, Calif...
http://www.kgi.edu/News-and-Events/FDA-Launches-Orphan-Drug-Workshop-at-KGI.html

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