Set up of experimental models of alkaptonuria and preclinical testing of therapeutic agents for the treatment of ochronotic arthropathy

  • 3 Years 2010/2013
  • 241.200€ Total Award
Alkaptonuria is a rare genetic disease with no cure. First asymptomatic, at the age of 40 severe articular problems may occur. We developed experimental models to test new drugs and propose the pre-clinical trial with acetylcysteine, a drug whose safety is guaranteed by 40 years of clinical use, as well as other types of drugs. This therapeutic could also prevent the onset of symptoms in genetically affected children.

Scientific Publications

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