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Reason for request
Variations to conditions for inclusion
-
Clinical Benefit
| Substantial |
Substantial |
Clinical Added Value
| important |
In isovaleric or methylmalonic or propionic organic acidaemias, during the initial acute episode and subsequent episodes of de compensation with hyperammonaemia, treatment with carglumic acid (CARBAGLU 200 mg, dispersible tablet or scored dispersible tablet) instituted as swiftly as possible as first-line therapy, usually as a component of an overall treatment strategy aimed at quickly and efficiently normalizing blood ammonia levels, offers a substantial improvement in actual benefit(level II) in the treatment strategy. |
