- |||||||||| Relyvrio (phenylbutyrate/taurursodiol) / Amylyx
Journal: Addendum: Relyvrio withdrawn. (Pubmed Central) - Jun 21, 2024 Initial findings suggest that this approach is safe. No abstract available
- |||||||||| sodium phenylbutyrate / Generic mfg.
Review, Journal: New insights into sodium phenylbutyrate as a pharmacotherapeutic option for neurological disorders. (Pubmed Central) - May 31, 2024 The study of new pharmacological options for the treatment of ND should focus on improving the characteristic symptoms and attacking specific molecular targets that allow the delay of damage processes such as neuroinflammation, oxidative stress, cellular metabolic dysfunction, and deregulation of transcriptional processes. In this review, we describe the possible role of sodium phenylbutyrate (NaPB) in the pathogenesis of Alzheimer's disease, hepatic encephalopathy, aging, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis; in addition, we describe the mechanism of action of NaPB and its beneficial effects that have been shown in various in vivo and in vitro studies to delay the evolution of any ND.
- |||||||||| SB225002 / GSK
Journal: CXCR2, as a key regulatory gene of HDP-PG-1, maintains intestinal mucosal homeostasis. (Pubmed Central) - May 5, 2024 The project aims to explore the regulatory mechanism of protegrin-1 (PG-1) expression using sodium phenylbutyrate (PBA) by omics sequencing technology and further investigate the role of key regulatory genes on intestinal health...Pre-treatment cells with a CXCR2 inhibitor (SB225002) effectively suppressed the induction of PG-1 by PBA...To that end, a gene involved in the HDP expression, CXCR2, has been identified in the study, which contributes to improving intestinal immune function. PBA may be used as a functional additive to regulate intestinal mucosal function, thereby enhancing the health of the intestinal and host.
- |||||||||| riluzole / Generic mfg., sodium phenylbutyrate / Generic mfg.
Racial/Ethnic Differences in Healthcare Resource Utilization Among Patients With Amyotrophic Lateral Sclerosis in Texas () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2007; In terms of ALS medication use, 202 (18.2%) patients were prescribed riluzole, 66 (5.9%) dextromethorphan HBr/quinidine sulfate, 24 (2.2%) edaravone, and 11 (1.0%) sodium phenylbutyrate/taurursodiol. Racial/ethnic differences exist in HCRU, with non-Hispanic Black and Hispanic patients having higher average number of ED visits per month and Hispanic patients having higher average hospital LOS.
- |||||||||| Breztri Aerosphere (budesonide/formoterol fumarate/glycopyrronium) / AstraZeneca
Characterization of PB&TURSO Use in the ALS ARC Database () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_353; This will provide a robust source for ongoing real-world study of PB&TURSO effectiveness and overall ALS treatment patterns . Concomitant use of PB&TURSO with other ALS medications was common among PLWALS.
- |||||||||| riluzole / Generic mfg., l-methylfolate / Generic mfg., sodium phenylbutyrate / Generic mfg.
Benefits of Early Combination Treatment in Patients with Amyotrophic Lateral Sclerosis: A Case Series (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_3533; This small ALS case series demonstrates that although combination therapy is feasible, there are significant limitations in drug obtainment. This retrospective pilot study provides preliminary evidence that the most benefit is derived from implementing combination treatment and warrants further investigation.
- |||||||||| rofecoxib (TRM-201) / Tremeau Pharma
Journal: Inferring Drug Set and Identifying the Mechanism of Drugs for PC3. (Pubmed Central) - Jan 27, 2024 We investigated how drugs affect drugs and how drugs affect genes in human pancreatic cancer cell lines that were derived from bone metastases of grade IV prostate cancer. Finally, we identified significant drug interactions within or between clusters, such as estradiol-rosiglitazone, estradiol-diclofenac, troglitazone-rosiglitazone, celecoxib-rofecoxib, celecoxib-diclofenac, and sodium phenylbutyrate-valproic acid.
- |||||||||| sodium phenylbutyrate / Generic mfg.
Trial completion date, Trial initiation date, Trial primary completion date: Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) (clinicaltrials.gov) - Jan 8, 2024 P2, N=24, Recruiting, Finally, we identified significant drug interactions within or between clusters, such as estradiol-rosiglitazone, estradiol-diclofenac, troglitazone-rosiglitazone, celecoxib-rofecoxib, celecoxib-diclofenac, and sodium phenylbutyrate-valproic acid. Trial completion date: Jun 2025 --> Dec 2025 | Initiation date: Dec 2023 --> Apr 2024 | Trial primary completion date: Dec 2024 --> Jun 2025
- |||||||||| Qalsody (tofersen) / Biogen, Tudcabil (tauroursodeoxycholic acid) / Bruschettini
Journal: Amyotrophic Lateral Sclerosis and Other Motor Neuron Diseases. (Pubmed Central) - Oct 22, 2023 An oral formulation of edaravone and an antisense oligonucleotide to a SOD1 gene variation (tofersen) have also recently been approved by the US Food and Drug Administration (FDA)...However, we still lack a definitive biomarker or a treatment that can halt the progression or reverse the course of disease. The evolving understanding of the genetic and pathophysiologic underpinnings of disease offers promise for more effective and clinically meaningful treatments in the future.
- |||||||||| Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
Journal: Treatment and management for children with urea cycle disorder in chronic stage. (Pubmed Central) - Oct 9, 2023 Gene therapy, stem cell therapy, enzyme therapy and other novel technologies may offer options for treatment in UCD patients. The regular biochemical assessments like blood ammonia, liver function and plasma amino acid profile are needed, and physical growth, intellectual development, nutritional intake should be also evaluated for adjusting treatment in time.
- |||||||||| Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
Journal: Monitoring the treatment of urea cycle disorders using phenylbutyrate metabolite analyses: Still many lessons to learn. (Pubmed Central) - Sep 17, 2023 Medications that elicit an alternate pathway for nitrogen excretion such as oral sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB) and intravenous sodium phenylacetate (NaPAA) are important for the management of urea cycle disorders (UCDs)...These results demonstrate that during routine clinical management, the majority of patients have PAA levels that are deemed safe. As age is negatively associated with PAA levels however, children undergoing treatment with NaPBA may need close monitoring of their phenylbutyrate metabolite levels.
- |||||||||| Qalsody (tofersen) / Biogen
Journal, HEOR: Managed care considerations to improve health care utilization for patients with ALS. (Pubmed Central) - Jul 15, 2023 Long-term studies have shown PB/TURSO to have a dual benefit on survival and function. The Institute for Clinical and Economic Review (ICER) 2022 Evidence Report for ALS does not value the high price points of edaravone and PB/TURSO as cost-effective based on the current evidence, despite valuing the need for new treatment options for this patient population.
- |||||||||| Qalsody (tofersen) / Biogen
Journal, HEOR: Optimizing pharmacologic treatment for ALS to improve outcomes and quality of life. (Pubmed Central) - Jul 15, 2023 Just 3 disease-modifying treatments-edaravone, riluzole, and sodium phenylbutyrate and taurursodiol (PB/TURSO)-are currently FDA approved to slow progression of amyotrophic lateral sclerosis (ALS)...Therapy selection is based largely upon patient characteristics, as guidelines have not been updated since the recent approval of PB/TURSO or accelerated approval of tofersen...Emerging agents offer some hope for patients with ALS. Among the drugs, biologics, and interventions under investigation for ALS are an oral tyrosine kinase inhibitor, RIPK1 inhibition, the use of mesenchymal stem cells, antisense oligonucleotides, sequential administration of all experimental treatments in a new study design, and modification of the patient's own mesenchymal stem cells.
- |||||||||| sodium phenylbutyrate / Generic mfg.
Journal: Sodium Phenylbutyrate/Taurursodiol. (Pubmed Central) - Jul 14, 2023 Among the drugs, biologics, and interventions under investigation for ALS are an oral tyrosine kinase inhibitor, RIPK1 inhibition, the use of mesenchymal stem cells, antisense oligonucleotides, sequential administration of all experimental treatments in a new study design, and modification of the patient's own mesenchymal stem cells. No abstract available
- |||||||||| Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
Urea cycle disorders in Argentina, analysis from a cohort of 135 patients diagnosed in the last 20 years. (Exhibitions Hall (ICC)) - Jul 11, 2023 - Abstract #SSIEM2023SSIEM_716; 76% required chronic treatment: sodium benzoate 76%, sodium phenylbutyrate 27%, and glycerol phenylbutyrate 12%...The mortality rate was higher than the one reported in other countries. We reported their presenting symptoms and sociocultural characteristics as well as accessibility to diagnosis and treatment, trying to identify the negative correlations in order to portray which variables are most related to worse results.
- |||||||||| riluzole / Generic mfg., sodium phenylbutyrate / Generic mfg.
Review, Journal: Current State and Future Directions in the Therapy of ALS. (Pubmed Central) - Jun 14, 2023 In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine.
- |||||||||| Relyvrio (phenylbutyrate/taurursodiol) / Amylyx
Review, Journal: Small molecules targeting different cellular pathologies for the treatment of amyotrophic lateral sclerosis. (Pubmed Central) - May 27, 2023 Compounds used to target cellular pathologies that stem from these mutations are discussed and comparisons among different preclinical models are presented. Because the drug discovery landscape for ALS and other motor neuron diseases is changing rapidly, we also offer recommendations for a novel, more effective, direction in ALS drug discovery that could accelerate translation of effective compounds from animals to patients.
- |||||||||| sodium phenylbutyrate / Generic mfg.
Journal: Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders. (Pubmed Central) - Apr 25, 2023 Lower systemic exposure of phenylacetate (PAA) and phenylbutyrate (PBA) was observed when ACER-001 was administered with a high-fat meal relative to a fasting state suggesting that the lower doses of PBA administered under fasting conditions may yield similar efficacy with potentially fewer dose dependent adverse effects relative to higher doses with a meal. ACER-001 appeared to be adequately taste-masked, staying below the aversive taste threshold for the first 3
- |||||||||| riluzole / Generic mfg., sodium phenylbutyrate / Generic mfg.
Idea Exchange: Amyotrophic Lateral Sclerosis (ALS) Pharmacotherapy and the Pharmacist (Learning Center) - Apr 2, 2023 - Abstract #AAPP2023AAPP_51; Prior to edaravone, the only FDA approved treatment option for treatment of ALS was riluzole...We will also discuss supportive therapies for management of mood, spasticity, and sialorrhea. In addition to discussing current treatment approaches, we plan to discuss opportunities for psychiatric pharmacists to utilize their unique skillset to assist in the management of ALS in an interdisciplinary setting.
- |||||||||| sodium phenylbutyrate / Generic mfg.
Journal: Sodium Phenylbutyrate and Taurursodiol. (Pubmed Central) - Mar 9, 2023 The survival benefit attributed to PB&TURSO in this analysis aligns with prior analyses using statistical models controlling for placebo-to-active crossover in CENTAUR. No abstract available
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