sodium phenylbutyrate / Generic mfg. 
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  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Preclinical, Journal:  Regulatory role of endoplasmic reticulum resident chaperone protein ERp29 in anti-murine ?-coronavirus host cell response. (Pubmed Central) -  Feb 27, 2023   
    Treatment of MHV-A59-infected astrocytes with the chemical chaperone 4-sodium phenylbutyrate increased ERp29 expression, rescued Cx43 transport to the cell surface, increased GJIC, and reduced ER stress...Treatment with Cx43 mimetic peptides inhibited GJIC and increased viral susceptibility, demonstrating a role for intercellular communication in reducing MHV-A59 infectivity. Taken together, these results support a therapeutically targetable ERp29-dependent mechanism where ?-coronavirus infectivity is modulated by reducing ER stress and rescuing Cx43 trafficking and function.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    SEVERE HYPERAMMONEMIA CAUSED BY CARNITINE DEFICIENCY DURING ADULTHOOD () -  Jan 25, 2023 - Abstract #SCCM2023SCCM_1235;    
    The patient was dialyzed and started on arginine, sodium benzoate/phenylacetate, citrulline, and buphenyl...Secondary carnitine deficiency is seen in severe malnutrition, and causes hyperammonemia and encephalopathy. This case is particularly important to note, as fatty acid oxidation disorders based on pure dietary deficiency that presents during adulthood are uncommon and not frequently considered in the differentials.
  • ||||||||||  Relyvrio (phenylbutyrate/taurursodiol) / Amylyx
    Journal:  New Drug Approved For ALS. (Pubmed Central) -  Dec 23, 2022   
    Sodium phenylbutyrate-taurursodiol (Relyvrio) has been approved to treat amyotrophic lateral sclerosis.The drug was approved despite the absence of phase 3 clinical trial data. Data from a small phase 2 trial indicated that patients receiving Relyvrio had a slower loss of functioning than those taking placebo.Relyvrio poses a risk to patients who are sensitive to salt intake and to those with pancreatic, intestinal, and enterohepatic circulation disorders.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Cerebrospinal Fluid Biomarker Effects From a Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol in Alzheimer’s Disease: Results From the PEGASUS Trial (Grand Ballroom AB - Tower 2) -  Dec 9, 2022 - Abstract #CTAD2022CTAD_343;    
    P2
    Results from PEGASUS provide the first-in-human evidence for a treatment effect of PB and TURSO on AD pathology and pathways of inflammation, synaptic function, oxidative stress, and neurodegeneration, complementing preclinical studies that showed a biologic effect for PB and TURSO both individually and in combination in AD All abstracts are embargoed until the day and time of presentation at the CTAD Conference S48 models. Taken together, these findings may be used to inform the design of subsequent trials and provide support for further clinical development of PB and TURSO for AD and other neurodegenerative diseases.
  • ||||||||||  Relyvrio (phenylbutyrate/taurursodiol) / Amylyx
    Journal:  Relyvrio for ALS. (Pubmed Central) -  Nov 19, 2022   
    Taken together, these findings may be used to inform the design of subsequent trials and provide support for further clinical development of PB and TURSO for AD and other neurodegenerative diseases. No abstract available
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Review, Journal:  ALSUntangled #64: butyrates. (Pubmed Central) -  Nov 16, 2022   
    Butyrates appear reasonably safe for use in humans. Based on the above information, we support a trial of a butyrate in PALS, but we cannot yet recommend one as a treatment.
  • ||||||||||  Relyvrio (phenylbutyrate/taurursodiol) / Amylyx
    Journal:  Effects of PB-TURSO on the transcriptional and metabolic landscape of sporadic ALS fibroblasts. (Pubmed Central) -  Oct 12, 2022   
    This study is the first to explore the molecular effects of Combo in ALS patient-derived cells. It shows that Combo has a greater and distinct impact compared with the individual compounds and provides clues to drug targets and mechanisms of action, which may underlie the benefits of this investigational drug combination.
  • ||||||||||  Relyvrio (phenylbutyrate/taurursodiol) / Amylyx
    Review, Journal:  Pharmacotherapy for Amyotrophic Lateral Sclerosis: A Review of Approved and Upcoming Agents. (Pubmed Central) -  Sep 29, 2022   
    Riluzole, a neuroprotective agent offering only a modest survival benefit, has long been the sole disease-modifying therapy for ALS...Sodium phenylbutyrate and taurursodiol (PB-TURSO) was conditionally approved in Canada in 2022, having shown significant slowing of disease progression and prolonged survival...This rich and ever-growing pipeline of investigational products, along with innovative clinical trial designs, collaborative trial networks, and an engaged ALS community', provide renewed hope to finding a cure for ALS. This article reviews existing ALS therapies and the current clinical drug development pipeline.
  • ||||||||||  riluzole / Generic mfg., sodium phenylbutyrate / Generic mfg.
    Journal:  Gold Coast Criteria Expand Clinical Trial Eligibility in Amyotrophic Lateral Sclerosis. (Pubmed Central) -  Sep 21, 2022   
    Until robust biomarkers are available to diagnose ALS, consensus diagnostic criteria remain necessary. Gold Coast criteria would expand research and clinical trial eligibility and improve external validity of clinical trial results.
  • ||||||||||  Albrioza (phenylbutyrate/taurursodiol) / Amylyx
    Review, Journal:  Sodium Phenylbutyrate and Ursodoxicoltaurine: First Approval. (Pubmed Central) -  Sep 20, 2022   
    The approval was based on results from the multicentre, phase II CENTAUR trial, in which slowing of ALS progression was demonstrated with sodium phenylbutyrate/ursodoxicoltaurine relative to placebo. This article summarizes the milestones in the development of sodium phenylbutyrate/ursodoxicoltaurine leading to this first approval.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    Clinical, Journal:  Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain. (Pubmed Central) -  Sep 13, 2022   
    This article summarizes the milestones in the development of sodium phenylbutyrate/ursodoxicoltaurine leading to this first approval. This study demonstrates the safety of direct switching from other nitrogen scavengers to GPB in clinical practice, which improves efficacy, metabolic control, and RAE compared to previous treatments.
  • ||||||||||  tofersen (BIIB067) / Biogen
    Journal:  Clinical studies in amyotrophic lateral sclerosis. (Pubmed Central) -  Sep 10, 2022   
    A phase III study with tofersen was negative but nevertheless yielded promising results. Important developments regarding the design of clinical ALS studies include the implementation of neurofilament light chain (NfL) levels as a standard outcome parameter and the consideration of progression rate for therapeutic response and stratification.
  • ||||||||||  fezagepras (PBI-4050) / Liminal BioSci
    Enrollment change, Trial termination:  A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate (clinicaltrials.gov) -  Aug 1, 2022   
    P1,  N=8, Terminated, 
    Abstract is embargoed at this time. N=16 --> 8 | Recruiting --> Terminated; Sponsor has decided to discontinue development of fezegepras based on initial pharmacokinetic results
  • ||||||||||  fezagepras (PBI-4050) / Liminal BioSci
    Enrollment open:  A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate (clinicaltrials.gov) -  May 24, 2022   
    P1,  N=16, Recruiting, 
    These data establish the proof-of-concept in humans that modulating the BCAA oxidative pathway may represent a potential treatment strategy for patients with T2D. Not yet recruiting --> Recruiting
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Journal:  Hastening the Diagnosis of Amyotrophic Lateral Sclerosis. (Pubmed Central) -  May 18, 2022   
    Such effects are observed in treatment with rilzuole, edaravone, methycobalamin, and sodium phenylbutyrate-taurursodiol in patient care and clinical trial settings...Challenges, however, still exist as to how to facilitate earlier recognition of possible ALS by primary care physicians and other non-neurologist providers, and how to foster a sense of urgency among neurologists to accelerate the process of diagnostic process. Herein we provide a number of recommendations that we hope will help achieve these ends.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Preclinical, Journal:  Gap Junction Protein, Connexin 43 is a Key Regulator of Metastasis in Syngeneic Ovarian Cancer Mouse Model. (Pubmed Central) -  May 14, 2022   
    A series of biochemical and cellular studies revealed that Sodium Phenylbutyrate(4PBA) treatment induced differential upregulation of Cx43 trafficking to the cell surface, rearrangement of cytoskeletal proteins, cell migration, and the formation of GJIC in ID8 and ID8-VEGF cells...Overall, The increased capacity of GJIC formation post-4PBA treatment is associated with reduction in cell migration in vitro (ID8 and ID8-VEGF) and tumor formation in vivo (ID8-VEGF). Interactome studies combining RNASeq and IPA are designed to understand the nexus between several cellular pathways, including oxidative stress in the regulation of Cx43 in the context of VEGF, to dissect the cellular and molecular mechanism of metastasis.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Journal:  Phenylbutyrate, a branched-chain amino acid (BCAA) keto dehydrogenase activator, promotes BCAA metabolism and induces muscle catabolism in C2C12 cells. (Pubmed Central) -  Apr 1, 2022   
    The compound sodium phenylbutyrate (PB) has been shown to promote branched-chain amino acid (BCAA) catabolism, and as such has been proposed as a treatment for disorders with enhanced BCAA levels: does PB induce muscle protein catabolism by forcing BCAA degradation away from muscle protein synthesis and mechanistic target of rapamycin (mTOR) inhibition?...Regulators of anabolic pathways mammalian target of rapamycin (mTOR) and its downstream components were impaired with PB treatment. The present results indicate that accelerated BCAA catabolism using PB resulted in adverse effects related to mTOR signalling and muscle protein metabolism, which may limit its application in settings where muscle wasting is a risk.
  • ||||||||||  SP600125 / BMS
    Journal:  Bisphenol A induces Agrp gene expression in hypothalamic neurons through a mechanism involving ATF3. (Pubmed Central) -  Jan 27, 2022   
    PBA treatment also regulates pro-inflammatory cytokine expression by inhibiting the transcriptional activation of NFκB-p65 and HDAC3 in SCs in vitro. Overall, these results indicate hypothalamic Agrp is susceptible to dysregulation by BPA and implicate ATF3 as a common mediator of the orexigenic effects of BPA in hypothalamic neurons.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Journal:  Diastereocontrol in Radical Addition to β-Benzyloxy Hydrazones: Revised Approach to Tubuvaline and Synthesis of O-Benzyltubulysin V Benzyl Ester. (Pubmed Central) -  Jan 27, 2022   
    To improve efficiency in this synthetic route to tubulysins, and to address difficulties in oxidation of the C-terminal alcohol, here we present two alternative routes to Tuv that (a) improve step economy, (b) provide modified conditions for Mn-mediated radical addition in the presence of aromatic heterocycles, and (c) expose an example of double diastereocontrol in radical addition to a β-benzyloxyhydrazone with broader implications for asymmetric amine synthesis via radical addition. An efficient coupling sequence affords 11-O-benzyltubulysin V benzyl ester.
  • ||||||||||  miglustat / Generic mfg.
    Clinical, Retrospective data, Journal, Orphan drug:  Clinical experience with orphan drugs for rare metabolic diseases. (Pubmed Central) -  Jan 27, 2022   
    These drugs have a high economic impact. The cost-effectiveness ratio for orphan drugs is a controversial issue due to their high cost and the inconclusive clinical evidence.
  • ||||||||||  lorazepam / Generic mfg.
    Clinical, Journal:  Ornithine Transcarbamylase Deficiency Presenting with Symptoms of Mania in a Young Adult Male. (Pubmed Central) -  Dec 30, 2021   
    In conclusion, partial ornithine transcarbamylase deficiency may manifest with psychiatric symptoms in early adulthood. In young patients with elevated ammonia and mental status change, OTCD is an important diagnosis to consider, as it is the most common inherited cause of hyperammonemia.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Review, Journal:  Synthetic Approaches Towards the Total synthesis of tubulysin and its fragments: A review. (Pubmed Central) -  Dec 27, 2021   
    A range of synthetic pathways adopted for the total synthesis of tubulysins and their fragments have been described in this review. Synthesis of fragments, Tuv, Tup, and Tut can be accomplished by adopting appropriate strategies such as Manganese-mediated synthesis, Ireland-Claisen rearrangement, Mukaiyama aldol reaction, and Mannich process etc. Tubulysin B, D, U, V, and N14-desacetoxytubulysin H have been prepared through Mitsunobu reaction, tert-butanesulfinamide method, Tandem reaction, aza-Barbier reaction, Evans aldol reaction, and C-H activation strategies etc. The remarkable anticancer potential of tubulysins toward a substantiate target make them prominent leads for developing novel drugs against multidrug-resistant cancers.
  • ||||||||||  lactulose / Generic mfg., sodium phenylbutyrate / Generic mfg.
    Clinical, Journal:  Differences in faecal microbiome composition between adult patients with UCD and PKU and healthy control subjects. (Pubmed Central) -  Sep 18, 2021   
    The differences in the microbiome composition of UCD patients compared to healthy controls were in part related to lactulose use...We propose as a first next step, to study the impact of these faecal microbiome alterations on metabolic stability. The faecal microbiome of UCD patients was less diverse compared to PKU patients and even more compared to healthy controls.