"Sodium phenylbutyrate" from_date:2012

Sodium phenylbutyrate EMA/123532/2013 EMEA/H/C/002500 EPAR summary for the public Pheburane sodium phenylbutyrate This is a summary of the European public assessment report (EPAR) for Pheburane. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Pheburane. For practical information about using Pheburane, patients should read the package leaflet or contact their doctor or pharmacist. What is Pheburane and what is it used for? Pheburane is a medicine that contains the active substance sodium phenylbutyrate. It is used to treat patients who have urea cycle disorders. These patients are not able to get rid of waste nitrogen from the body because they lack some enzymes

Sodium phenylbutyrate, Ursodoxicoltaurine (Albrioza) Terms of use - Canada.ca * Skip to main content * Skip to "About government" Language selection * Français SearchSearch Canada.ca Search Topics menuMain Menu You are here: 1. Home 2. Health 3. Drug and health products 4. Licensing, authorizing and manufacturing drug and health products 5. Drug and health product review and approval 6. Clinical

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Sodium phenylbutyrate/taurursodiol (Relyvrio) - To treat amyotrophic lateral sclerosis (ALS) Skip to main contentSkip to FDA SearchSkip to footer links An official website of the United States government Here's how you know U.S. Food and Drug Administration  Search   MenuSearch FDASubmit search Home Drugs Drug Approvals and Databases Drugs@FDADrug Approval Package

sodium phenylbutyrate and ursodoxicoltaurine - Albrioza - Amyotrophic lateral sclerosis (ALS) Skip to main contentAboutCollaboration/OutreachPatient/CommunityCareersContactMy CADTHFRReportsResourcesProvide InputSubmit a RequestNews & EventsWhat Does The Evidence Say About...SearchBreadcrumbHome Reimbursement Reviews sodium phenylbutyrate and ursodoxicoltaurineCopied to clipboard sodium phenylbutyrate and ursodoxicoltaurine( Last Updated : October 24, 2022)Reimbursement ReviewAbout CADTH Reimbursement ReviewsProcedures and TemplatesFiling an ApplicationProcess OverviewCADTH Provisional Funding AlgorithmsCADTH Pharmaceutical Review UpdateClinician Input InstructionsOpen CallsPatient Input InstructionsAdvisory CommitteesParticipating Drug ProgramsCOVID-19 ResponseGeneric Name: sodium

An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis. Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, fatal neurodegenerative motor neuron disease. Despite the overwhelming need for effective therapeutics for ALS, riluzole and edaravone were the only two FDA-approved disease-modifying therapies prior to 2022 . The randomized, double-blind, multicenter, placebo-controlled CENTAUR trial demonstrated the safety and efficacy of sodium phenylbutyrate-taurursodiol (PB-TURSO) in persons with ALS (PALS), leading to its conditional approval in Canada in June 2022 and full approval in the USA in September 2022. Herein, the authors provide a review of the pharmacology and clinical trials evaluating sodium phenylbutyrate

Effect of sodium phenylbutyrate and taurursodiol on plasma concentrations of neuroinflammatory biomarkers in amyotrophic lateral sclerosis: results from the CENTAUR trial. An oral sodium phenylbutyrate and taurursodiol combination (PB and TURSO) significantly reduced functional decline in people living with amyotrophic lateral sclerosis (ALS) in the CENTAUR trial. Biomarkers linking clinical

Analysis of sodium phenylbutyrate and taurursodiol survival effect in ALS using external controls. Sodium phenylbutyrate and taurursodiol (PB and TURSO) was evaluated in amyotrophic lateral sclerosis (ALS) in the CENTAUR trial encompassing randomized placebo-controlled and open-label extension phases. On intent-to-treat (ITT) survival analysis, median overall survival (OS) was 4.8 months longer

Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial. Coformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) was shown to prolong survival and slow functional decline in amyotrophic lateral sclerosis (ALS). Determine whether PB/TURSO prolonged tracheostomy

Sodium Phenylbutyrate Rescues Thyroid Hormone Transport in Brain Endothelial-Like Cells. Monocarboxylate transporter 8 (MCT8) deficiency is a rare genetic disease leading to a severe developmental delay due to a lack of thyroid hormones (THs) during critical stages of human brain development. Some MCT8-deficient patients are not as severely affected as others. Previously, we hypothesized that these patients' mutations do not affect the functionality but destabilize the MCT8 protein, leading to a diminished number of functional MCT8 molecules at the cell surface. We have already demonstrated that the chemical chaperone sodium phenylbutyrate (NaPB) rescues the function of these mutants by stabilizing their protein expression in an overexpressing cell system. Here, we expanded our previous work

Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis. Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy and safety of a combination of the two compounds in persons with amyotrophic lateral sclerosis (ALS) are not known. In this multicenter, randomized, double-blind trial, we enrolled participants with definite ALS who had had an onset of symptoms within the previous 18 months. Participants were randomly assigned in a 2:1 ratio to receive sodium phenylbutyrate-taurursodiol (3 g of sodium phenylbutyrate and 1 g of taurursodiol, administered once a day for 3 weeks and then twice a day) or placebo. The primary outcome was the rate of decline in the total score on the Amyotrophic Lateral Sclerosis

Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis. An orally administered, fixed-dose coformulation of sodium phenylbutyrate-taurursodiol (PB-TURSO) significantly slowed functional decline in a randomized, placebo-controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long-term survival analysis

Sodium Phenylbutyrate An official website of the United States government Here's how you know Log inAccess keysNCBI HomepageMyNCBI HomepageMain ContentMain NavigationBookshelfSearch databaseBooksAll DatabasesAssemblyBiocollectionsBioProjectBioSampleBooksClinVarConserved DomainsdbGaPdbVarGeneGenomeGEO DataSetsGEO ProfilesGTRHomoloGeneIdentical Protein GroupsMedGenMeSHNLM Levels and EffectsSummary of Use during LactationNo information is available on the clinical use of sodium phenylbutyrate or the combination of sodium phenylbutyrate and taurursodiol during breastfeeding. Both sodium phenylbutyrate and taurursodiol are highly protein bound and therefore unlikely to enter milk in clinically important amounts. If sodium phenylbutyrate with or without taurursodiol

Pheburane - sodium phenylbutyrate 21 February 2013 EMA/212039/2012 Committee for Medicinal Products for Human Use (CHMP) Assessment report Pheburane International non-proprietary name: sodium phenylbutyrate Procedure No. EMEA/H/C/002500 Assessment Report as adopted by the CHMP with all information of a commercially confidential nature deleted. 7 Westferry Circus ● Canary Wharf of the overall data submitted and the scientific discussion within the Committee, issued a positive opinion for granting a Marketing Authorisation to Pheburane on 21 February 2013. Pheburane Assessment report Page 6/24 2. Scientific discussion 2.1. Introduction Pheburane is a medicinal product that contains sodium phenylbutyrate as active substance. It is intended for oral administration

Efficacy of orally administered sodium benzoate and sodium phenylbutyrate in dogs with congenital portosystemic shunts. Hyperammonemia can result in hepatic encephalopathy, which in severe cases eventually can lead to coma and death. In dogs, congenital portosystemic shunts (CPSS) are the most common cause for hyperammonemia. Conservative treatment consists of a protein modified diet , nonabsorbable disaccharides, antibiotics, or some combinations of these. Sodium benzoate (SB) and sodium phenylbutyrate (SPB) both are used in the acute and long-term treatment of humans with hyperammonemia caused by urea cycle enzyme deficiencies. Both treatments are believed to lower blood ammonia concentrations by promoting excretion of excess nitrogen via alternative pathways. To evaluate the efficacy

Sodium phenylbutyrate (Pheburane®) Sodium phenylbutyrate (Pheburane® ) ..

Sodium phenylbutyrate (Pheburane) - treatment of active Systemic Juvenile Idiopathic Arthritis (SJIA)

The chaperone-like sodium phenylbutyrate improves factor IX intracellular trafficking and activity impaired by the frequent p.R294Q mutation. Essentials Missense mutations often impair protein folding, and thus intracellular trafficking and secretion. Cellular models of severe type I hemophilia B were challenged with chaperone-like compounds. Sodium phenylbutyrate improved intracellular

The effect of combined treatment with sodium phenylbutyrate and cisplatin, erlotinib, or gefitinib on resistant NSCLC cells Chemotherapy resistance is the main cause of the marginal clinical benefit of platinum-based chemotherapy and tyrosine kinase inhibitors in advanced non-small-cell lung cancer (NSCLC). Thus, the identification of new therapeutic agents that can enhance the sensitivity of these drugs is of clinical importance. Histone deacetylase inhibitors (HDACIs) are emerging as new promising agents with strong antiproliferative effects against different types of cancers. This study investigates the synergistic potential of sodium phenylbutyrate (NaPB) added on top of standard chemotherapy used against NSCLC. The objective of this study was to evaluate the ability of NaPB to overcome

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A\>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD. Participation in the study will require three outpatient visits