The management of organic acidaemias: The role of transplantation

  title={The management of organic acidaemias: The role of transplantation},
  author={James V. Leonard and John H. Walter and Patrick J Mckiernan},
  journal={Journal of Inherited Metabolic Disease},
This workshop addressed the issue of treatment in propionic acid-aemia (PA) and methylmalonic acidaemia (MMA) and in particular the outcome of conventional management compared with organ transplantation. Although it appears that with medical treatment the mortality for early-onset disease has improved, long-term outcome remains poor. However, liver and liver/kidney transplantation is associated with a high perioperative mortality and long-term complications. It offers only a partial cure. The… CONTINUE READING

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