Reason for request
Inclusion
Clinical Benefit
| Substantial |
The actual benefit of PHEBURANE is considerable.
|
Clinical Added Value
| minor |
In view of : - the pharmaceutical formulation, which is able to mask the unpleasant taste of sodium phenylbutyrate - the clinical follow-up data of 17 patients treated with PHEBURANE under the terms of a temporary usage authorisation (TUA), who showed fewer episodes of vomiting and hyperammonaemic decompensation on PHEBURANE by comparison with AMMONAPS, PHEBURANE offers a minor improvement in actual benefit (IAB IV) over AMMONAPS as adjunctive therapy in the long-term management of urea cycle disorders involving deficiency in carbamoyl phosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.
|
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
VU8Z8ELtAT2Udetq