Seen from elsewhere – Ongoing reforms in the EU aim to promote the use of drugs already authorized for new pathologies. A solution to accelerate treatment approval and reduce research costs.
By Oriol Guell (El Pace)
Nick Sirau was getting ready to change his newborn son’s diaper when he found a large, dark red spot. It was a Sunday in October in the year 2000, and the then 27-year-old’s life was about to change forever. “The emergency doctor came, but they found nothing. He said it could be due to the red cabbage my wife ate, which can pass into breast milk and then urine. We only found out later that he had alkaptonuria.”
, he recalls. It is a rare genetic disease, from which Nick Sirau’s second son also suffers, and which manifests itself by darkening certain spinal tissues and arthritis.Almost 20 years later, on September 17, 2020, the European Medicines Agency (EMA) authorized the first treatment of alkaptonuria with a drug called nitisinone, which had been used for many years. …
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