Apr 14, 2020

Many researchers, both in the public and private sectors, are working to bring novel pharmaceuticals to tyrosinemia patients.  Nitisinone is the pharmaceutical of choice, and there has been a lot of work done on some specific aspects of tyrosinemia, such as the behavioral and psychological consequences of the disease. We are excited to report a major potential advance in the development of pharmaceuticals for the treatment of tyrosinemia. Codexis, a California-based company, has applied for a patent for a possible tyrosinemia protein scavenger. This means that there is the potential for a drug that would “harvest” excess tyrosine in the body and help eliminate it. This drug is not yet available on the market, and it still probably requires many challenging years to develop such a drug, but this is a very important development. A similar drug has been developed recently for a related disease called phenylketonuria, and so there is great hope and excitement that a similar drug will be successful for tyrosinemia. We will keep the community posted on this very important development.

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