Page last updated: 2024-11-02

4-phenylbutyric acid and Orphan Diseases

4-phenylbutyric acid has been researched along with Orphan Diseases in 2 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.

Orphan Diseases: Rare diseases that have not been well studied.

Research Excerpts

ExcerptRelevanceReference
" 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)

Research

Studies (2)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's1 (50.00)24.3611
2020's1 (50.00)2.80

Authors

AuthorsStudies
Cederbaum, SD1
Edwards, J1
Kellmeyer, T1
Peters, Y1
Steiner, RD1
Vanhoorne, V1
Peeters, E1
Van Tongelen, I1
Boussery, K1
Wynendaele, E1
De Spiegeleer, B1
Remon, JP1
Vervaet, C1

Other Studies

2 other studies available for 4-phenylbutyric acid and Orphan Diseases

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
Pharmaceutical compounding of orphan active ingredients in Belgium: how community and hospital pharmacists can address the needs of patients with rare diseases.
    Orphanet journal of rare diseases, 2019, 08-01, Volume: 14, Issue:1

    Topics: Arginine; Belgium; Carnitine; Drug Compounding; Excipients; Humans; Pharmacists; Phenylbutyrates; Pr

2019