buphenyl

buphenyl

An orphan drug for managing urea cycle disorders—e.g., deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase—and indicated for infants with neonatal-onset deficiencies presenting within the first 28 days of life, in whom episodes of acute hyperammonemic encephalopathy are often fatal, and when not, often result in profound mental retardation.

Adverse effects
Amenorrhoea, anorexia, body odour due to phenylacetate excess, abdominal pain, nausea, arrhythmias, renal tubular acidosis, rash, headaches, syncope, weight gain, metabolic acidosis, increased alkaline phosphatase, anaemia.
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References in periodicals archive ?
The company is expected to beat on the back of sales from Ravicti and Buphenyl, both for the treatment of urea cycle disorders.
The prescription medication is Buphenyl, which is sodium phenylbutyrate.
Her test tube studies suggest that a drug called buphenyl could guide the protein to where it belongs.
Hyperion isn't the first company to make a cure for the rare disease, but it is much more convenient than the existing option, Ucycld Pharma's Buphenyl.
BUPHENYL is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
Full Prescribing Information for BUPHENYL is available at http://www.
Subjects were administered BUPHENYL or glycerol phenylbutyrate at equimolar doses (i.
At least one adverse event (AE) was reported by 23 subjects during BUPHENYL treatment and 27 subjects during glycerol phenylbutyrate treatment.
Patients were enrolled on their stable dose of BUPHENYL and 24-hour PK, ammonia measurements, amino acids, and urine collections were completed in a study unit after seven days of on-study treatment.
Twenty-one adverse events (AEs) occurred in seven subjects during BUPHENYL treatment and fifteen AEs occurred in five subjects with HPN-100.
5 teaspoons) delivers the same amount of active ingredient as the maximum daily dose of BUPHENYL (forty tablets or 6.
sales and marketing efforts of the only two FDA-approved compounds for the treatment of UCD, AMMONUL and BUPHENYL.