Details for Patent: 9,962,358
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Title: | Methods of therapeutic monitoring of nitrogen scavenging drugs |
Abstract: | The present disclosure provides methods for evaluating daily ammonia exposure based on a single fasting ammonia blood level measurement, as well as methods that utilize this technique to adjust the dosage of a nitrogen scavenging drug, determine whether to administer a nitrogen scavenging drug, and treat nitrogen retention disorders. |
Inventor(s): | Scharschmidt; Bruce (San Francisco, CA), Mokhtarani; Masoud (Walnut Creek, CA) |
Assignee: | Horizon Therapeutics, LLC (Lake Forest, IL) |
Filing Date: | Aug 25, 2017 |
Application Number: | 15/687,118 |
Claims: | 1. A method of treating a urea cycle disorder (UCD) in a pediatric subject in need thereof wherein the pediatric subject has experienced repeated episodes of hyperammonemia and/or chronically elevated ammonia which have impaired the pediatric subject's neurocognitive function, comprising: administering glyceryl tri-[4-phenylbutyrate] (HPN-100) to the pediatric subject in an amount sufficient to maintain the pediatric subject's fasting plasma ammonia level at less than half the upper limit of normal, wherein such administration improves neurocognitive function in the pediatric subject. 2. The method of claim 1, wherein administration of HPN-100 reduces total ammonia burden. 3. The method of claim 1, wherein administration of HPN-100 reverses the pediatric subject's neurocognitive impairment. 4. The method of claim 1, wherein neurocognitive function is assessed by BRIEF (Behavior Rating Inventory of Executive Function). 5. The method of claim 1, wherein the pediatric subject has neonatal presentation of the UCD. 6. The method of claim 1, wherein the pediatric subject has intercurrent illness. |