Except where otherwise noted, this work is distributed under the terms of a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International licence (CC BY-NC-ND), a copy of which is available at http://creativecommons.org/licenses/by-nc-nd/4.0/
NCBI Bookshelf. A service of the National Library of Medicine, National Institutes of Health.
Glycerol Phenylbutyrate (Ravicti) [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2017 Apr.
- 1.
- The physician’s guide to urea cycle disorders [Internet]. Danbury (CT): National Organization for Rare Disorders; 2012. [cited 2016 Nov 17]. (NORD guides for physicians). Available from: http:
//nordphysicianguides .org/wp-content /uploads/2012/02/NORD _Physician_Guide_to _Urea_Cycle_Disorders.pdf - 2.
- Mew NA, Lanpher BC, Gropman A, Chapman KA, Simpson KL, Urea Cycle Disorders Consortium, et al. Urea cycle disorders overview. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 2015 Apr 9 [cited 2016 Nov 17]. Available from: https://www
.ncbi.nlm .nih.gov/books/NBK1217/ [PMC free article: PMC1217] [PubMed: 20301396] - 3.
- Lee B. Urea cycle disorders: clinical features and diagnosis. In: Post TW, editor. UpToDate [Internet]. Waltham (MA): UpToDate; 2015 Oct 21 [cited 2016 Nov 17]. Available from: www
.uptodate.com Subscription required. - 4.
- Martin-Hernandez E, Alamiz-Echevarria L, Castejon-Ponce E, Pedron-Giner C, Couce ML, Serrano-Nieto J, et al. Urea cycle disorders in Spain: an observational, cross-sectional and multicentric study of 104 cases. Orphanet J Rare Dis [Internet]. 2014 Nov 30 [cited 2016 Nov 17];9:187. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC4258263 [PMC free article: PMC4258263] [PubMed: 25433810] - 5.
- Summar ML, Koelker S, Freedenberg D, Le Mons C, Haberle J, Lee H-S, et al. The incidence of urea cycle disorders. Mol Genet Metab [Internet]. 2013 [cited 2016 Nov 17];110(11):179–80. Available from: https://www
.ncbi.nlm .nih.gov/pmc/articles/PMC4364413/ - 6.
- Births (84F0210X) [Internet]. Ottawa (ON): Statistics Canada; 2009. Highlights. [cited 2016 Nov 17]. Available from: http://www
.statcan.gc .ca/pub/84f0210x/2009000 /aftertoc-aprestdm1-eng.htm - 7.
- Batshaw ML, Tuchman M, Summar M, Seminara J, Members of the Urea Cycle Disorders Consortium. A longitudinal study of urea cycle disorders. Mol Genet Metab [Internet]. 2014 Sep [cited 2016 Nov 17];113(1–2):127–30. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC4178008 [PMC free article: PMC4178008] [PubMed: 25135652] - 8.
- Haberle J, Boddaert N, Burlina A, Chakrapani A, Dixon M, Huemer M, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis [Internet]. 2012 May 29 [cited 2016 Nov 17];7:32. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC3488504 [PMC free article: PMC3488504] [PubMed: 22642880] - 9.
- Krivitzky L, Babikian T, Lee HS, Thomas NH, Burk-Paull KL, Batshaw ML. Intellectual, adaptive, and behavioral functioning in children with urea cycle disorders. Pediatr Res [Internet]. 2009 Jul [cited 2016 Nov 17];66(1):96–101. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC2746951 [PMC free article: PMC2746951] [PubMed: 19287347] - 10.
- Ah Mew N, Krivitzky L, McCarter R, Batshaw M, Tuchman M, Urea Cycle Disorders Consortium of the Rare Diseases Clinical Research Network. Clinical outcomes of neonatal onset proximal versus distal urea cycle disorders do not differ. J Pediatr [Internet]. 2013 Feb [cited 2016 Nov 17];162(2):324–9. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC4440324 [PMC free article: PMC4440324] [PubMed: 22901741] - 11.
- Urea Cycle Disorders Consortium. Urea cycle treatment guidelines [Internet]. Washington (DC): Rare Diseases Clinical Research Network; 2016. [cited 2016 Nov 17]. Available from: http://www
.rarediseasesnetwork .org/cms/ucdc /Healthcare-Professionals /Urea-Cycle-Treatment-Guidelines - 12.
- Lee B. Urea cycle disorders: management. In: Post TW, editor. UpToDate [Internet]. Waltham (MA): UpToDate; 2016 Feb 8 [cited 2016 Oct 19]. Available from: www
.uptodate.com Subscription required. - 13.
- Batshaw ML, Brusilow S, Waber L, Blom W, Brubakk AM, Burton BK, et al. Treatment of inborn errors of urea synthesis: activation of alternative pathways of waste nitrogen synthesis and excretion. N Engl J Med. 1982 Jun 10;306(23):1387–92. [PubMed: 7078580]
- 14.
- Brusilow SW, Maestri NE. Urea cycle disorders: diagnosis, pathophysiology, and therapy. Adv Pediatr. 1996;43:127–70. [PubMed: 8794176]
- 15.
- Batshaw ML, MacArthur RB, Tuchman M. Alternative pathway therapy for urea cycle disorders: twenty years later. J Pediatr. 2001 Jan;138(1 Suppl):S46–S54. [PubMed: 11148549]
- 16.
- PrRavicti™ (glycerol phenylbutyrate): oral liquid, 1.1 g/mL [product monograph]. Dublin: Horizon Pharma Ireland Ltd.; 2016 Mar 16.
- 17.
- PrRavicti: summary basis of decision [Internet]. Ottawa (ON): Health Canada; 2016 Jul 7. [cited 2016 Nov 17]. Available from: http://www
.hc-sc.gc.ca /dhp-mps/prodpharma /sbd-smd/drug-med/sbd-smd-2016-ravicti-174219-eng.php - 18.
- PrPheburane® (sodium phenylbutyrate granules): 483 mg per gram of granules [product monograph]. Blainville (QC): Médunik Canada; 2015 Jan 23.
- 19.
- Diaz GA, Krivitzky LS, Mokhtarani M, Rhead W, Bartley J, Feigenbaum A, et al. Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate. Hepatology [Internet]. 2013 Jun [cited 2016 Oct 13];57(6):2171–9. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC3557606 [PMC free article: PMC3557606] [PubMed: 22961727] - 20.
- Center for Drug Evaluation and Research, U.S. Food and Drug Administration. Medical review(s). In: Ravicti (glycerol phenylbutyrate) oral liquid. Company: Hyperion Therapeutics Inc. Application no.: 203284. Approval date: 2/01/2013 [Internet]. Rockville (MD): FDA; 2013 Feb 1 [cited 2016 Aug 29]. (FDA drug approval package). Available from: http://www
.accessdata .fda.gov/drugsatfda_docs /nda/2013/203284Orig1s000TOC.cfm - 21.
- Center for Drug Evaluation and Research, U.S. Food and Drug Administration. Statistical review(s). In: Ravicti (glycerol phenylbutyrate) oral liquid. Company: Hyperion Therapeutics Inc. Application no.: 203284. Approval date: 2/01/2013 [Internet]. Rockville (MD): FDA; 2013 Feb 1 [cited 2016 Aug 29]. (FDA drug approval package). Available from: http://www
.accessdata .fda.gov/drugsatfda_docs /nda/2013/203284Orig1s000TOC.cfm - 22.
- Committee for Medicinal Products for Human Use. Assessment report: Ravicti [Internet]. London: European Medicines Agency; 2015 Sep 24. [cited 2016 Oct 19]. (EPAR - Public assessment report). Available from: http://www
.ema.europa .eu/docs/en_GB/document_library /EPAR_-_Public _assessment_report /human/003822/WC500199159.pdf - 23.
- CDR submission: Ravicti™, glycerol phenylbutyrate, 1.1 g/mL, oral liquid. Company: Horizon Therapeutics Canada [CONFIDENTIAL manufacturer’s submission]. Ottawa (ON): Horizon Therapeutics Canada; 2016 Aug 29.
- 24.
- Clinical Study Report: HPN-100-006. 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 [CONFIDENTIAL internal manufacturer’s report]. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2011 May 13.
- 25.
- McGuire BM, Zupanets IA, Lowe ME, Xiao X, Syplyviy VA, Monteleone J, et al. Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis. Hepatology [Internet]. 2010 Jun [cited 2016 Dec 9];51(6):2077–85. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC3733097 [PMC free article: PMC3733097] [PubMed: 20512995] - 26.
- Lee B, Diaz GA, Rhead W, Lichter-Konecki U, Feigenbaum A, Berry SA, et al. Blood ammonia and glutamine as predictors of hyperammonemic crises in patients with urea cycle disorder. Genet Med. 2015 Jul;17(7):561–8. [PMC free article: PMC4465427] [PubMed: 25503497]
- 27.
- Lee B, Diaz GA, Rhead W, Lichter-Konecki U, Feigenbaum A, Berry SA, et al. Glutamine and hyperammonemic crises in patients with urea cycle disorders. Mol Genet Metab [Internet]. 2016 Jan [cited 2016 Oct 13];117(1):27–32. Available from: https://www
.ncbi.nlm .nih.gov/pmc/articles /PMC4915945/pdf/nihms794360.pdf [PMC free article: PMC4915945] [PubMed: 26586473] - 28.
- Bachmann C. Outcome and survival of 88 patients with urea cycle disorders: a retrospective evaluation. Eur J Pediatr. 2003 Jun;162(6):410–6. [PubMed: 12684900]
- 29.
- Uchino T, Endo F, Matsuda I. Neurodevelopmental outcome of long-term therapy of urea cycle disorders in Japan. J Inherit Metab Dis. 1998;21 Suppl 1:151–9. [PubMed: 9686352]
- 30.
- Msall M, Batshaw ML, Suss R, Brusilow SW, Mellits ED. Neurologic outcome in children with inborn errors of urea synthesis. Outcome of urea-cycle enzymopathies. N Engl J Med. 1984 Jun 7;310(23):1500–5. [PubMed: 6717540]
- 31.
- Kido J, Nakamura K, Mitsubuchi H, Ohura T, Takayanagi M, Matsuo M, et al. Long-term outcome and intervention of urea cycle disorders in Japan. J Inherit Metab Dis. 2012 Sep;35(5):777–85. [PubMed: 22167275]
- 32.
- Clinical Study Report: HPN-100-005. 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 [CONFIDENTIAL internal manufacturer’s report]. Version 2.0. Amendment 1. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2011 May 24.
- 33.
- Clinical Study Report: HPN-100-007. A phase 3, open-label study of the safety of HPN-100 for the long-term treatment of urea cycle disorders [CONFIDENTIAL internal manufacturer’s report]. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2011 Nov 30.
- 34.
- Clinical Study Report (Safety Extension): HPN-100-005. 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 [CONFIDENTIAL internal manufacturer’s report]. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2011 Nov 30.
- 35.
- Mapi Research Trust, Varni JW. Scaling and scoring of the pediatric quality of life inventory™. PedsQL™ [Internet]. Boston (MA): Mapi Research Trust; 2014 Mar. 130 p. [cited 2016 Dec 1]. Available from: http://www
.pedsql.org/PedsQL-Scoring.pdf - 36.
- Ware J, Kosinski M, Bjorner JB, Turner-Bowker DM, Gandek B, Maruish ME. Determining important differences in scores. In: User’s manual for the SF-36v2 health survey. 2nd ed. Lincoln (RI): Quality Metric Inc.; 2007.
- 37.
- Maccow G. Overview of WASI-II [slide deck]. San Antonio (TX): Pearson Clinical; 2011. Available from: http://images
.pearsonclinical .com/images /PDF/Webinar/WASI-IIHandoutOct2011.pdf - 38.
- Pearson Clinical [Internet]. San Antonio (TX): Pearson Clinical. Wechsler Abbreviated Scale of Intelligence™; 1999 [cited 2016 Dec 1]. Available from: http://www
.pearsonclinical .com/education /products/100000593/wechsler-abbreviated-scale-of-intelligence-wasi.html - 39.
- Clinical Study Report: HPN-100-012. A switch-over, open-label study of the safety, pharmacokinetics, and efficacy of HPN-100, in pedatric subjects under 6 years of age with urea cycle disorders (UCDs) [CONFIDENTIAL internal manufacturer’s report]. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2012 Apr 6.
- 40.
- Clinical Study Report (Safety Extension): HPN-100-012. A switch-over, open-label study of the safety, pharmacokinetics, and efficacy of HPN-100, in pedatric subjects under 6 years of age with urea cycle disorders (UCDs) [CONFIDENTIAL internal manufacturer’s report]. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2013 Dec 19.
- 41.
- Goertzel B. The Wechsler preschool and primary scale of intelligence (WPPSI): a brief overview [[Internet]]. Rockville (MD): Ben Goertzel; 2013 Feb 13. [cited 2016 Dec 1]. Available from: http://goertzel
.org/Preschool _IQ_Test_Overview.pdf - 42.
- Pearson Clinical [Internet]. San Antonio (TX): Pearson Clinical. Wechsler Preschool and Primary Scale of Intelligence™ - Fourth Edition (WPPSI™ - IV); 2012 [cited 2016 Dec 1]. Available from: http://www
.pearsonclinical .com/psychology /products/100000102 /wechsler-preschool-and-primary-scale-of-intelligence--fourth-edition-wppsi-iv .html - 43.
- Lafayette Instrument® Evaluation. Products [Internet]. Lafayette (IN): Lafayette Instrument Company. Grooved pegboard test. Model 32025; 2012 [cited 2016 Dec 1]. Available from: http:
//lafayetteevaluation .com/product_detail.asp?itemid=167 - 44.
- Lafayette Instrument® Evaluation. Products. Lafayette (IN): Lafayette Instrument. Grooved pegboard test. User instructions. Model 32025; 2002 Available from: https:
//lafayetteevaluation .com/downloads2.asp?catid=2 - 45.
- Woods DL, Kishiyama MM, Yund EW, Herron TJ, Edwards B, Poliva O, et al. Improving digit span assessment of short-term verbal memory. J Clin Exp Neuropsychol [Internet]. 2011 [cited 2016 Dec 1];33(1):101–11. Available from: https://www
.ncbi.nlm .nih.gov/pmc/articles /PMC2978794/pdf/nihms208687.pdf [PMC free article: PMC2978794] [PubMed: 20680884] - 46.
- National Longitudinal Surveys [Internet]. Washington (DC): NLS. National Longitudinal Survey of youth. Children and young adults; 2012? [cited 2016 Dec 1]. Available from: https://www
.nlsinfo.org /content/cohorts/nlsy79-children /topical-guide /assessments /wechsler-intelligence-scale-children - 47.
- Pearson Clinical [Internet]. San Antonio (TX): Pearson Clinical. California Verbal Learning Test® - Second Edition (CVLT® - II); 2000 [cited 2016 Dec 1]. Available from: http://www
.pearsonclinical .com/psychology /products/100000166 /california-verbal-learning-test--second-edition-cvlt-ii .html#tab-details - 48.
- Woods SP, Delis DC, Cobb Scott J, Kramer JH, Holdnack JA. The California Verbal Learning Test - second edition: Test-retest reliability, practice effects, and reliable change indices for the standard and alternate forms. Arch Clin Neuropsychol [Internet]. 2006 Aug [cited 2016 Dec 1];21(5):413–20. Available from: http://www
.sciencedirect .com/science/article /pii/S0887617706000606 [PubMed: 16843636] - 49.
- Manual for the Assessment Data Manager program (ADM) for the CBCL/4–18, YSR, TRF, YASR, YABCL, CBCL/2–3, CBCL/12–5 & C-TRF [Internet]. Burlington (VT): Achenback System of Empirically Based Assessment (ASEBA); 2000 Oct. Available from: http://www
.aseba.org /products/manuals/ADM2.pdf - 50.
- Mazefsky CA, Anderson R, Conner CM, Minshew N. Child Behavior Checklist scores for school-aged children with Autism: preliminary evidence of patterns suggesting the need for referral. J Psychopathol Behav Assess [Internet]. 2011 Mar [cited 2016 Dec 1];33(1):31–7. Available from: https://www
.ncbi.nlm .nih.gov/pmc/articles /PMC3362998/pdf/nihms256967.pdf [PMC free article: PMC3362998] [PubMed: 22661827] - 51.
- PAR [Internet]. Lutz (FL): PAR, Inc. Behavior Rating Inventory of Executive Function® (BRIEF®); 2012 [cited 2016 Dec 1]. Available from: http://www4
.parinc.com/Products/Product .aspx?ProductID=BRIEF - 52.
- Gioia GA, Isquith PK, Guy SC, Kenworthy L. (BRIEF®): Behavior Rating Inventory of Executive Function® [slide deck on Internet]. Lutz (FL): PAR, Inc.; 2016 Jan 12. [cited 2016 Dec 1]. Available from: http://www4
.parinc.com /WebUploads/samplerpts/BRIEF.ppt - 53.
- Isquith PK, Gioia GA, PAR staff. (BRIEF®): Behavior Rating Inventory of Executive Function®. Parent form score report [Internet]. Lutz (FL): PAR, Inc.; 2011. [cited 2016 Dec 1]. Available from: http://www4
.parinc.com /WebUploads/samplerpts /BRIEF_Parent_Score_PiC.pdf - 54.
- Isquith PK, Gioia GA, PAR staff. (BRIEF®): Behavior Rating Inventory of Executive Function®. Teacher form score report [Internet]. Lutz (FL): PAR, Inc.; 2011. [cited 2016 Dec 1]. Available from: http://www4
.parinc.com /WebUploads/samplerpts /BRIEF_Teacher_Score_PiC.pdf - 55.
- Mahone EM, Cirino PT, Cutting LE, Cerrone PM, Hagelthorn KM, Hiemenz JR, et al. Validity of the behavior rating inventory of executive function in children with ADHD and/or Tourette syndrome. Arch Clin Neuropsychol [Internet]. 2002 Oct [cited 2016 Dec 1];17(7):643–62. Available from: http://www
.sciencedirect .com/science/article /pii/S0887617701001688 [PubMed: 14591848] - 56.
- Waisbren SE, He J, McCarter R. Assessing psychological functioning in metabolic disorders: validation of the Adaptive Behavior Assessment System, Second Edition (ABAS-II), and the Behavior Rating Inventory of Executive Function (BRIEF) for identification of individuals at risk. JIMD Rep [Internet]. 2015 [cited 2017 Jan 9];21:35–43. Available from: http://www
.ncbi.nlm.nih .gov/pmc/articles/PMC4470946 [PMC free article: PMC4470946] [PubMed: 25712381] - 57.
- Clinical Study Report: UP 1204-003. A phase 2, open-label, switch-over, dose-escalation study of the safety and tolerability of HPN-100 (glyceryl tri [4 phenylbutyrate]) compared to Buphenyl® (sodium phenylbutyrate) in patients with urea cycle disorders [CONFIDENTIAL internal manufacturer’s report]. Version 2.0. Amendment 1. South San Francisco (CA): Hyperion Therapeutics, Inc.; 2011 Jun 27.
- REFERENCES - Glycerol Phenylbutyrate (Ravicti)REFERENCES - Glycerol Phenylbutyrate (Ravicti)
- Executive Summary - Clinical Review Report: Cysteamine delayed-release capsules ...Executive Summary - Clinical Review Report: Cysteamine delayed-release capsules (Procysbi)
- ABBREVIATIONS - Somatropin (Genotropin) (0.15 mg/day to 0.3 mg/day)ABBREVIATIONS - Somatropin (Genotropin) (0.15 mg/day to 0.3 mg/day)
- Introduction - Clinical Review Report: Slexipag (Uptravi)Introduction - Clinical Review Report: Slexipag (Uptravi)
- Executive Summary - Clinical Review Report: Icosapent Ethyl (Vascepa)Executive Summary - Clinical Review Report: Icosapent Ethyl (Vascepa)
Your browsing activity is empty.
Activity recording is turned off.
See more...