NTBC treatment in tyrosinaemia type I: Long-term outcome in French patients

  title={NTBC treatment in tyrosinaemia type I: Long-term outcome in French patients},
  author={Alice Masurel-Paulet and Jos{\'e}phine Poggi-Bach and M.-O. Rolland and Olivier Bernard and Nathalie Guffon and Dries Dobbelaere and Jacques Sarles and H{\'e}l{\`e}ne Ogier de Baulny and Guy Touati},
  journal={Journal of Inherited Metabolic Disease},
We describe a retrospective study of long-term outcome of 46 patients treated and regularly followed in France with 2-(2-nitro-4-trifluoromethylbenzoyl)-1, 3-cyclohexanedione (NTBC) for tyrosinaemia type I. Most had initial good response with normalization of liver function and metabolic parameters. Only one infant had no response to treatment and required liver transplantation. Among the 45 long-term treated patients, three underwent secondary liver transplantation: one for cirrhosis and two… CONTINUE READING
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Outcome of tyrosinemia type III

  • CJ Ellaway, E Holme, S Standing
  • J Inherit Metab Dis
  • 2001
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