Compounds > 4-phenylbutyric acid
Page last updated: 2024-11-02

4-phenylbutyric acid and Urea Cycle Disorders, Inborn

4-phenylbutyric acid has been researched along with Urea Cycle Disorders, Inborn in 15 studies

4-phenylbutyric acid: RN refers to the parent cpd
4-phenylbutyric acid : A monocarboxylic acid the structure of which is that of butyric acid substituted with a phenyl group at C-4. It is a histone deacetylase inhibitor that displays anticancer activity. It inhibits cell proliferation, invasion and migration and induces apoptosis in glioma cells. It also inhibits protein isoprenylation, depletes plasma glutamine, increases production of foetal haemoglobin through transcriptional activation of the gamma-globin gene and affects hPPARgamma activation.

Urea Cycle Disorders, Inborn: Rare congenital metabolism disorders of the urea cycle. The disorders are due to mutations that result in complete (neonatal onset) or partial (childhood or adult onset) inactivity of an enzyme, involved in the urea cycle. Neonatal onset results in clinical features that include irritability, vomiting, lethargy, seizures, NEONATAL HYPOTONIA; RESPIRATORY ALKALOSIS; HYPERAMMONEMIA; coma, and death. Survivors of the neonatal onset and childhood/adult onset disorders share common risks for ENCEPHALOPATHIES, METABOLIC, INBORN; and RESPIRATORY ALKALOSIS due to HYPERAMMONEMIA.

Research Excerpts

ExcerptRelevanceReference
" All treated UCD patients were included in a follow-up protocol developed by the Laboratory (Lucane Pharma) and the French Medicines Agency (ANSM), which recorded demographics, dosing characteristics of NaPB, concomitant medications, adverse events, and clinical outcome during the period of treatment."2.79Results from a Nationwide Cohort Temporary Utilization Authorization (ATU) survey of patients in france treated with Pheburane(®) (Sodium Phenylbutyrate) taste-masked granules. ( Barth, M; Brassier, A; Dobbelaere, D; Guffon, N; Kibleur, Y, 2014)
" No statistically significant differences were observed in plasma phenylacetic acid and PAGN exposure during dosing with GPB vs."2.76Ammonia control in children with urea cycle disorders (UCDs); phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate. ( Beliveau, M; Diaz, GA; Dickinson, K; Feigenbaum, A; Jomphe, C; Lichter-Konecki, U; Marier, JF; Martinez, A; Mauney, J; Merritt, JL; Mokhtarani, M; Rhead, W; Scharschmidt, B, 2011)
" Adverse events were comparable for the two drugs; 2 subjects experienced hyperammonemic events on NaPBA while none occurred on GPB."2.75Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control. ( Beliveau, M; Berry, SA; Diaz, GA; Dickinson, K; Gargosky, S; Lee, B; Marier, JF; Martinez, A; Mauney, J; Mian, A; Mokhtarani, M; Rhead, W; Scharschmidt, BF; Shchelochkov, O, 2010)
" Data on demographics, dosing characteristics of NaPB, concomitant medications, adverse events and clinical outcomes were collected at a follow-up visit after 1-2 years of treatment with the drug administered under marketing conditions."2.53Long-Term Follow-Up on a Cohort Temporary Utilization Authorization (ATU) Survey of Patients Treated with Pheburane (Sodium Phenylbutyrate) Taste-Masked Granules. ( Guffon, N; Kibleur, Y, 2016)
"4-PBA is currently used clinically to treat urea cycle disorders under the trade name Buphenyl."2.52The therapeutic effects of 4-phenylbutyric acid in maintaining proteostasis. ( Ask, K; Ayaub, EA; Dickhout, JG; Kolb, PS; Yum, V; Zhou, W, 2015)
" Four Phase 1 studies were conducted to characterize the bioavailability (BA) and/or bioequivalence (BE) of ACER-001 (in healthy volunteers) and taste assessment relative to NaPBA powder (in taste panelists)."1.91Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders. ( Cederbaum, SD; Edwards, J; Kellmeyer, T; Peters, Y; Steiner, RD, 2023)
" Plasma concentrations of their primary metabolite, phenylacetate (PAA), as well as the ratio of PAA to phenylacetylglutamine (PAGN) are useful for guiding dosing and detecting toxicity."1.91Monitoring the treatment of urea cycle disorders using phenylbutyrate metabolite analyses: Still many lessons to learn. ( Burrage, LC; Elsea, SH; Glinton, KE; Liu, N; Minard, CG; Nagamani, SCS; Sun, Q, 2023)
" A more precise dosing device is provided with the coated granules than with the uncoated granules (Ammonaps)."1.42Sodium phenylbutyrate coated granules (Pheburane). Defective urea synthesis: a welcome formulation. ( , 2015)

Research

Studies (15)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's13 (86.67)24.3611
2020's2 (13.33)2.80

Authors

AuthorsStudies
Cederbaum, SD1
Edwards, J1
Kellmeyer, T1
Peters, Y1
Steiner, RD1
Glinton, KE1
Minard, CG1
Liu, N1
Sun, Q1
Elsea, SH1
Burrage, LC1
Nagamani, SCS1
Andrade, F1
Vitoria, I1
Martín Hernández, E1
Pintos-Morell, G1
Correcher, P1
Puig-Piña, R1
Quijada-Fraile, P1
Peña-Quintana, L1
Marquez, AM1
Villate, O1
García Silva, MT1
de Las Heras, J1
Ceberio, L1
Rodrigues, E1
Almeida Campos, T1
Yahyaoui, R1
Blasco, J1
Vives-Piñera, I1
Gil, D1
Del Toro, M1
Ruiz-Pons, M1
Cañedo, E1
Barba Romero, MA1
García-Jiménez, MC1
Aldámiz-Echevarría, L1
Yamasaki, K1
Enokida, T1
Taguchi, K1
Miyamura, S1
Kawai, A1
Miyamoto, S1
Maruyama, T1
Seo, H1
Otagiri, M1
Guha, M1
Kibleur, Y2
Dobbelaere, D1
Barth, M1
Brassier, A1
Guffon, N2
Kolb, PS1
Ayaub, EA1
Zhou, W1
Yum, V1
Dickhout, JG1
Ask, K1
Lee, B3
Rhead, W3
Diaz, GA4
Scharschmidt, BF3
Mian, A1
Shchelochkov, O1
Marier, JF2
Beliveau, M2
Mauney, J4
Dickinson, K4
Martinez, A2
Gargosky, S1
Mokhtarani, M4
Berry, SA3
Cederbaum, S2
Lemons, C3
Batshaw, ML1
Lichter-Konecki, U3
Merritt, JL2
Feigenbaum, A2
Jomphe, C1
Scharschmidt, B1
Krivitzky, LS1
Bartley, J1
Longo, N1
Berquist, W1
Gallagher, R1
Bartholomew, D1
Harding, CO2
McCandless, SE2
Smith, W2
Vockley, G1
Bart, SA1
Korson, MS1
Kronn, D1
Zori, R1
C S Nagamani, S1
Moors, TL1
Coakley, DF2
Mistry, PK1
Moors, T1

Clinical Trials (5)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children 6-17 Years of Age With Urea Cycle Disorders, With a Long-Term Safety Extension[NCT00947544]Phase 217 participants (Actual)Interventional2010-03-31Completed
A Phase 3, Randomized, Double-Blind, Cross-Over, Active-Controlled Study of the Efficacy and Safety of HPN-100, Glyceryl Tri-(4-phenylbutyrate), for the Treatment of Adults With Urea Cycle Disorders (Help UCD)[NCT00992459]Phase 346 participants (Actual)Interventional2009-10-31Completed
A Phase 3, Open-Label Study of the Safety of HPN-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)[NCT00947297]Phase 360 participants (Actual)Interventional2009-11-30Completed
A Phase 2, Open-Label, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 Compared to Buphenyl® (Sodium Phenylbutyrate) in Patients With Urea Cycle Disorders[NCT00551200]Phase 214 participants (Actual)Interventional2007-10-31Completed
A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment With HPN-100, in Pediatric Subjects Under 6 Years of Age With Urea Cycle Disorders (UCDs)[NCT01347073]Phase 323 participants (Actual)Interventional2011-07-31Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Average Ammonia Values on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug (Switch Over)

blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionµmol/L (Mean)
HPN-10028.68
NaPBA37.75

Blood Ammonia Control

To evaluate control of blood ammonia by HPN-100 compared with NaPBA in pediatric patients with UCDs. (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionμmol∙h/L (Mean)
HPN-100603.83
NaPBA814.62

NH3 Cmax on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug

blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionμmol/L (Mean)
HPN-10047.77
NaPBA55.66

Plasma PAA (Phenylacetate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug

blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionμg•h/mL AUC 0-24 (Mean)
HPN-100964
NaPBA773

Plasma PAGN AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug

blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionμg*h/mL AUC 0-24 (Mean)
HPN-1001378
NaPBA1015

Plasma PBA (Phenylbutyrate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug

blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionµg*h/ml AUC 0-24 (Mean)
HPN-100631
NaPBA236

Quality of Life Assessed by the SF-15 Questionnaire

"change from baseline to Month 12.~The SF 15 questionnaire consists of 15 questions that assess the following:~Physical functioning (5 questions)~Emotional functioning (4 questions)~Social functioning (3 questions)~School functioning (3 questions) Items were scored on a 5-point Likert scale from 0 (never) to 4 (almost always) or a 3-point scale (0 [not at all], 2 [sometimes], or 4 [a lot] for the young child self-report). Items were reverse-scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, and 4=0. Total score was 0-100 scale (averaged from each functional areas). In the 0-100 scale, 0 is the worst score and 100 is best score.~Improved quality of life was shown by increased total score from baseline to Month 12." (NCT00947544)
Timeframe: 1 year

Interventionscore on a scale (Mean)
HPN-1004.0

Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA vs. HPN-100

blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionpercentage of sample (Number)
HPN-10018.4
NaPBA31.6

Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events)

To evaluate the safety and PK characteristics of HPN-100 compared with sodium phenylbutyrate (NaPBA) in pediatric patients with urea cycle disorders (UCDs) (NCT00947544)
Timeframe: 1 week on each treatment for a total of 2 week.

Interventionparticipants (Number)
HPN-1004
NaPBA2

Urinary PAGN 24-hour Excretion Values on NaPBA vs. HPN-100 (Switch Over)

Urinary PAGN (phenylacetylglutamine) 24-hour excretion. Urine was collect during 0-12 hrs and 12-24 hrs. (NCT00947544)
Timeframe: Day 7 (NaPBA) and Day 14 (HPN-100)

Interventionμg (Mean)
HPN-10012501037
NaPBA12512426

Number and Causes of Hyperammonemic Events (Safety Extension)

"Number of Subjects with at Least One Hyperammonemic Crisis.~Hyperammonemic crisis is defined as follows:~• Clinical symptoms associated with ammonia of ≥ 100 µmol/L" (NCT00947544)
Timeframe: 1 year

,
Interventionparticipants (Number)
Number of subjects with at least 1 HACNumber of Crises
Pre-Enrollment (NaPBA)58
Safety Extension (HPN-100)33

Cmax for PAA of NaPBA and HPN-100 in Plasma

Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28

Interventionμg/mL (Mean)
NaPBA52.2
HPN-10038.5

Cmax for PBA of NaPBA and HPN-100 in Plasma

Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28

Interventionμg/mL (Mean)
NaPBA80.9
HPN-10051.9

Cmax PAGN of NaPBA and HPN-100 in Plasma

Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28

Interventionμg/mL (Mean)
NaPBA78.6
HPN-10086.8

Correlation Between Urinary Phenylacetylglutamine (PAGN) Excretion Over 24 Hours (U-PAGN24-hour Excr) and Venous Ammonia - Area Under the Concentration-time Curve From Time 0 (Predose) to 24 Hours (AUC0-24)

The correlation between 24-hour urinary phenylacetylglutamine (PAGN) excretion (U-PAGN24-hour Excr) and venous ammonia AUC0-24 was summarized and the correlation was tested using the Spearman rank-order correlation. (NCT00992459)
Timeframe: 28 Days

Interventioncorrelation coefficient (Number)
NaPBA0.437
HPN-1000.219

Maximum Ammonia Values Observed on NaPBA Versus HPN-100

Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28

Interventionµmol/L (Mean)
NaPBA70.83
HPN-10060.94

Number and Severity of Symptomatic Hyperammonemic Crises

Severity of symptomatic hyperammonemic crises was measured by peak ammonia level (µmol/L) when it is >= 100 µmol/L. (NCT00992459)
Timeframe: 29 Days

Interventionevents (Number)
NaPBA1
HPN-1000

Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA Versus HPN-100

NaPBA treated arm: total 345 blood samples were collected. HPN-100 treated arm: 343 blood samples were collected. (NCT00992459)
Timeframe: on Day 14 and Day 28

Interventionsamples (Number)
NaPBA125
HPN-100122

Rate of Adverse Events in Each Treatment Group

(NCT00992459)
Timeframe: 29 Days

Interventionparticipants (Number)
NaPBA23
HPN-10027

The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28.

Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. Arm A day 14 and Arm B day 28 data were combined as a NaPBA treatment Arm. Arm B day 14 and Arm A day 28 data were combined as a HPN-100 treatment Arm. (NCT00992459)
Timeframe: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28

Interventionμmol∙h/L (Mean)
NaPBA976.6
HPN-100865.35

U-PAGN24-hour Excr of NaPBA and HPN-100

(NCT00992459)
Timeframe: 24 hours on Day 14 of each treatments

Interventionμg (Mean)
NaPBA13627515
HPN-10013502745

Number and Causes of Hyperammonemic Events

Number of hyperammonemic crises per patient (NCT00947297)
Timeframe: 1 year

Interventionhyperammonemic events (Mean)
HPN-1000.20

Patient Satisfaction With HPN-100

Drug preference will be noted at week 3 (NCT00947297)
Timeframe: Month 1 post dose

Intervention% preferred HPN-100 (Number)
HPN-10090

Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug)

(NCT00947297)
Timeframe: 1 year

Interventionparticipants (Number)
HPN-10033

Blood Ammonia Levels

Venous Ammonia levels over time (NCT00947297)
Timeframe: 1 Year

InterventionUmol/L (Mean)
BaselineMonth 12
HPN-10027.62324.202

Number of Subjects Experienced Adverse Events

(NCT00551200)
Timeframe: during the period on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks)

Interventionparticipants (Number)
Buphenyl7
HPN-1005

Number of Subjects Experienced Serious Adverse Events

(NCT00551200)
Timeframe: during the period subjects on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks)

Interventionparticipants (Number)
Buphenyl1
HPN-1000

Drug Preference for HPN-100 or Buphenyl® (as Assessed by Global Preference Question)

(NCT00551200)
Timeframe: End of Study

Interventionparticipants (Number)
prefer Buphenylprefer HPN-100
Buphenyl to HPN-10019

Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites)

measured AUC0-24 (Area under the curve from time 0 (pre-dose) to 24 hours) for each metabolite in plasma. Data were collected at 30 minutes and 1, 2, 4, 5, 6, 8, 10, 12, and 24 hours post-first dose. (NCT00551200)
Timeframe: At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone)

,
Interventionμg*h/mL (Mean)
AUC0-24 PBA (phenylbutyrate) in plasmaAUC0-24 PAA (phenylacetate) in plasmaAUC0-24 PAGN (phenylacetylglutamine) in plasma
HPN-100 Steady State5405751098
NaPBA Steady State7405961133

Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve)

Data were collected at pre-first dose and at 30 minutes and 1, 2, 4, 5, 6, 8, 10, 12, and 24 hours post first dose. (NCT00551200)
Timeframe: At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone), and at steady state (1 week) after each dose escalation

,
Interventionμmol/L (Mean)
in peakin TNAUC (time-normalized area under the curve)
HPN-100 Steady State56.326.5
NaPBA Steady State79.138.4

Adverse Events

Rate of adverse events during the Safety Extension portion of the protocol ( please note: HPN-100 treatment only during Safety Extension ) (NCT01347073)
Timeframe: 12 months

Interventionparticipants (Number)
HPN-10023

Adverse Events

Rate of adverse events during the Switch-Over portion of the Protocol (NCT01347073)
Timeframe: 2 weeks

Interventionparticipants (Number)
NaPBA0
HPN-1006

Blood Ammonia

24-hour ammonia AUC of blood ammonia levels on Days 1 (NaPBA) and 10 (HPN-100) were compared. Ammonia was assessed at Hour 0 (pre-first dose, fasted), Hour 8 (~2-4 hours after lunch or the second main meal and dose of NaPBA), Hour 12 (~4 hours after the last main meal) and 24 hours post-first dose (pre-first dose on following day, fasted). (NCT01347073)
Timeframe: 2 weeks

Interventionumol/L*hours (Mean)
NaPBA914.43
HPN-100647.63

Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA

Ammonia values were converted to SI units (umol/L) and normalized to a standard ULN of 35 umol/L prior to analysis (NCT01347073)
Timeframe: 2 weeks

InterventionAmmonia Values > ULN (Number)
NaPBA22
HPN-1008

Hyperammonemic Crisis

Rate of HAC during pre-enrollment on NaPBA compared to HAC during HPN-100 treatment (NCT01347073)
Timeframe: 1 year

Interventionnumber of crises (Number)
Pre-enrollment29
Long-term Phase12

Reviews

2 reviews available for 4-phenylbutyric acid and Urea Cycle Disorders, Inborn

ArticleYear
The therapeutic effects of 4-phenylbutyric acid in maintaining proteostasis.
    The international journal of biochemistry & cell biology, 2015, Volume: 61

    Topics: Animals; Endoplasmic Reticulum; Endoplasmic Reticulum Stress; Homeostasis; Humans; Phenylbutyrates;

2015
Long-Term Follow-Up on a Cohort Temporary Utilization Authorization (ATU) Survey of Patients Treated with Pheburane (Sodium Phenylbutyrate) Taste-Masked Granules.
    Paediatric drugs, 2016, Volume: 18, Issue:2

    Topics: Administration, Oral; Chemistry, Pharmaceutical; Follow-Up Studies; Health Care Surveys; Humans; Pat

2016

Trials

5 trials available for 4-phenylbutyric acid and Urea Cycle Disorders, Inborn

ArticleYear
Results from a Nationwide Cohort Temporary Utilization Authorization (ATU) survey of patients in france treated with Pheburane(®) (Sodium Phenylbutyrate) taste-masked granules.
    Paediatric drugs, 2014, Volume: 16, Issue:5

    Topics: Adolescent; Adult; Child; Child, Preschool; France; Humans; Male; Middle Aged; Phenylbutyrates; Qual

2014
Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control.
    Molecular genetics and metabolism, 2010, Volume: 100, Issue:3

    Topics: Adult; Aged; Ammonia; Cross-Over Studies; Female; Glutamine; Glycerol; Humans; Male; Middle Aged; Ph

2010
Ammonia control in children with urea cycle disorders (UCDs); phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate.
    Molecular genetics and metabolism, 2011, Volume: 103, Issue:4

    Topics: Adolescent; Ammonia; Child; Dose-Response Relationship, Drug; Glycerol; Humans; Male; Phenylbutyrate

2011
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Adolescent; Adult; Ammonia; Child; Cross-Over Studies; Double-Blind Method; Female; Glutamine; Glyce

2013
Ammonia control in children ages 2 months through 5 years with urea cycle disorders: comparison of sodium phenylbutyrate and glycerol phenylbutyrate.
    The Journal of pediatrics, 2013, Volume: 162, Issue:6

    Topics: Ammonia; Child; Child, Preschool; Drug Substitution; Female; Glutamine; Glycerol; Humans; Infant; Li

2013

Other Studies

8 other studies available for 4-phenylbutyric acid and Urea Cycle Disorders, Inborn

ArticleYear
Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders.
    Molecular genetics and metabolism, 2023, Volume: 138, Issue:4

    Topics: Humans; Hyperammonemia; Nitrogen; Phenylbutyrates; Powders; Rare Diseases; Taste; Urea; Urea Cycle D

2023
Monitoring the treatment of urea cycle disorders using phenylbutyrate metabolite analyses: Still many lessons to learn.
    Molecular genetics and metabolism, 2023, Volume: 140, Issue:3

    Topics: Child; Humans; Phenylbutyrates; Retrospective Studies; Urea Cycle Disorders, Inborn

2023
Quantification of urinary derivatives of Phenylbutyric and Benzoic acids by LC-MS/MS as treatment compliance biomarkers in Urea Cycle disorders.
    Journal of pharmaceutical and biomedical analysis, 2019, Nov-30, Volume: 176

    Topics: Adolescent; Adult; Benzoates; Biomarkers; Child; Child, Preschool; Chromatography, High Pressure Liq

2019
Species Differences in the Binding of Sodium 4-Phenylbutyrate to Serum Albumin.
    Journal of pharmaceutical sciences, 2017, Volume: 106, Issue:9

    Topics: Animals; Binding Sites; Cattle; Humans; Molecular Docking Simulation; Phenylbutyrates; Protein Bindi

2017
Urea cycle disorder drug approved.
    Nature biotechnology, 2013, Volume: 31, Issue:4

    Topics: Adult; Clinical Trials as Topic; Drug Approval; Drug Industry; Glycerol; Humans; Patient Compliance;

2013
Sodium phenylbutyrate coated granules (Pheburane). Defective urea synthesis: a welcome formulation.
    Prescrire international, 2015, Volume: 24, Issue:157

    Topics: Administration, Oral; Chemistry, Pharmaceutical; Drug Dosage Calculations; Humans; Phenylbutyrates;

2015
Alternative pathway or diversion therapy for urea cycle disorders now and in the future.
    Molecular genetics and metabolism, 2010, Volume: 100, Issue:3

    Topics: Animals; Drug Approval; History, 20th Century; History, 21st Century; Humans; Phenylbutyrates; Unite

2010
Rare disease clinical research network's urea cycle consortium delivers a successful clinical trial to improve alternate pathway therapy.
    Hepatology (Baltimore, Md.), 2013, Volume: 57, Issue:6

    Topics: Ammonia; Female; Glycerol; Humans; Male; Phenylbutyrates; Urea Cycle Disorders, Inborn

2013