Carbamoyl Phosphate Synthetase Deficiency
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EMA approval · 2025-01-14EMA Authorised: Pheburane (sodium phenylbutyrate) — Eurocept International B. V.
The EMA authorised Pheburane (sodium phenylbutyrate) for treating carbamoyl-phosphate synthetase I deficiency, with decision on 17 December …