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Opinions on drugs - Posted on May 15 2014

Reason for request

Variations to conditions for inclusion


Clinical Benefit



Clinical Added Value

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