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  Subjects -> PHARMACY AND PHARMACOLOGY (Total: 575 journals)
Showing 1 - 200 of 253 Journals sorted alphabetically
AAPS Journal     Hybrid Journal   (Followers: 31)
AAPS Open     Open Access   (Followers: 5)
AAPS PharmSciTech     Hybrid Journal   (Followers: 6)
AboutOpen     Open Access  
ACS Pharmacology & Translational Science     Hybrid Journal   (Followers: 5)
Acta Pharmaceutica     Open Access   (Followers: 4)
Acta Pharmaceutica Indonesia     Open Access  
Acta Pharmaceutica Sinica B     Open Access   (Followers: 1)
Acta Pharmacologica Sinica     Hybrid Journal   (Followers: 3)
Acta Physiologica Hungarica     Full-text available via subscription  
Actualites Pharmaceutiques     Full-text available via subscription   (Followers: 4)
Advanced Drug Delivery Reviews     Hybrid Journal   (Followers: 98)
Advanced Herbal Medicine     Open Access   (Followers: 9)
Advanced Therapeutics     Hybrid Journal   (Followers: 1)
Advances in Medical, Pharmaceutical and Dental Research     Open Access   (Followers: 5)
Advances in Pharmacoepidemiology & Drug Safety     Open Access   (Followers: 3)
Advances in Pharmacological and Pharmaceutical Sciences     Open Access   (Followers: 11)
Advances in Pharmacology     Full-text available via subscription   (Followers: 21)
Advances in Pharmacology and Pharmacy     Open Access   (Followers: 9)
Advances in Traditional Medicine     Hybrid Journal   (Followers: 4)
Adverse Drug Reaction Bulletin     Full-text available via subscription   (Followers: 5)
AJP : The Australian Journal of Pharmacy     Full-text available via subscription   (Followers: 10)
Al-Azhar Journal of Pharmaceutical Sciences     Open Access   (Followers: 3)
Alternatives to Laboratory Animals     Full-text available via subscription   (Followers: 9)
American Journal of Cardiovascular Drugs     Hybrid Journal   (Followers: 20)
American Journal of Drug Discovery and Development     Open Access   (Followers: 3)
American Journal of Health-System Pharmacy     Full-text available via subscription   (Followers: 59)
American Journal of Pharmacological Sciences     Open Access   (Followers: 2)
American Journal of Pharmacology and Toxicology     Open Access   (Followers: 24)
American Journal of Therapeutics     Hybrid Journal   (Followers: 13)
Analytical Methods     Hybrid Journal   (Followers: 8)
Annales Pharmaceutiques Francaises     Full-text available via subscription  
Annals of Pharmacotherapy     Hybrid Journal   (Followers: 56)
Annual Review of Pharmacology and Toxicology     Full-text available via subscription   (Followers: 38)
Anti-Infective Agents     Hybrid Journal   (Followers: 5)
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry     Hybrid Journal   (Followers: 6)
Antibiotics     Open Access   (Followers: 12)
Antibody Therapeutics     Open Access  
Antiviral Chemistry and Chemotherapy     Open Access   (Followers: 1)
Antiviral Research     Hybrid Journal   (Followers: 8)
Applied Clinical Trials     Full-text available via subscription   (Followers: 7)
Archiv der Pharmazie     Hybrid Journal   (Followers: 2)
Archives of Drug Information     Hybrid Journal   (Followers: 4)
Archives of Pharmacal Research     Full-text available via subscription   (Followers: 2)
Archives of Pharmacy and Pharmaceutical Sciences     Open Access   (Followers: 2)
Archives of Razi Institute     Open Access  
Archivos Venezolanos de Farmacología y Terapéutica     Open Access  
Ars Pharmaceutica     Open Access  
Asian Journal of Medical and Pharmaceutical Researches     Open Access  
Asian Journal of Pharmaceutical Research and Health Care     Open Access   (Followers: 2)
Asian Journal of Pharmaceutical Sciences     Open Access   (Followers: 1)
Asian Journal of Pharmaceutics     Open Access   (Followers: 1)
Asian Journal of Research in Medical and Pharmaceutical Sciences     Open Access  
ASSAY and Drug Development Technologies     Hybrid Journal   (Followers: 3)
Australian Journal of Herbal Medicine     Full-text available via subscription   (Followers: 4)
Australian Pharmacist     Full-text available via subscription   (Followers: 7)
Autonomic & Autacoid Pharmacology     Hybrid Journal  
Avicenna Journal of Phytomedicine     Open Access   (Followers: 1)
Bangladesh Journal of Pharmacology     Open Access  
Bangladesh Journal of Physiology and Pharmacology     Open Access  
Bangladesh Pharmaceutical Journal     Full-text available via subscription  
Basic & Clinical Pharmacology & Toxicology     Hybrid Journal   (Followers: 15)
Behavioural Pharmacology     Hybrid Journal   (Followers: 2)
Bioanalysis     Full-text available via subscription   (Followers: 11)
Biochemical Pharmacology     Hybrid Journal   (Followers: 10)
BioDrugs     Full-text available via subscription   (Followers: 8)
Biological & Pharmaceutical Bulletin     Full-text available via subscription   (Followers: 3)
Biomarkers in Drug Development     Partially Free   (Followers: 2)
Biomaterials     Hybrid Journal   (Followers: 55)
Biomedical and Environmental Sciences     Full-text available via subscription   (Followers: 2)
Biomedicine & Pharmacotherapy     Full-text available via subscription   (Followers: 2)
Biometrical Journal     Hybrid Journal   (Followers: 9)
Biopharm International     Full-text available via subscription   (Followers: 20)
Biopharmaceutics and Drug Disposition     Hybrid Journal   (Followers: 11)
BMC Pharmacology     Open Access   (Followers: 3)
BMC Pharmacology & Toxicology     Open Access   (Followers: 9)
Brazilian Journal of Pharmaceutical Sciences     Open Access   (Followers: 1)
British Journal of Clinical Pharmacology     Hybrid Journal   (Followers: 31)
British Journal of Pharmacology     Hybrid Journal   (Followers: 17)
British Journal of Pharmacy (BJPharm)     Open Access   (Followers: 2)
Bulletin of Faculty of Pharmacy, Cairo University     Open Access   (Followers: 2)
CADTH Technology Overviews     Free  
Canadian Journal of Pain     Open Access   (Followers: 3)
Canadian Journal of Physiology and Pharmacology     Hybrid Journal   (Followers: 2)
Canadian Pharmacists Journal / Revue des Pharmaciens du Canada     Hybrid Journal   (Followers: 3)
Cancer Biotherapy & Radiopharmaceuticals     Hybrid Journal  
Cancer Chemotherapy and Pharmacology     Hybrid Journal   (Followers: 4)
Cardiovascular Drugs and Therapy     Hybrid Journal   (Followers: 14)
Cardiovascular Therapeutics     Open Access   (Followers: 3)
Cephalalgia Reports     Open Access  
Chemical and Pharmaceutical Bulletin     Full-text available via subscription   (Followers: 1)
Chemical Research in Toxicology     Hybrid Journal   (Followers: 22)
ChemMedChem     Hybrid Journal   (Followers: 9)
Chemotherapy     Full-text available via subscription   (Followers: 3)
Chinese Herbal Medicines     Full-text available via subscription   (Followers: 1)
Chinese Journal of Pharmaceutical Analysis     Full-text available via subscription  
Ciencia e Investigación     Open Access  
Ciência Equatorial     Open Access  
Clinical and Experimental Pharmacology and Physiology     Hybrid Journal   (Followers: 7)
Clinical and Translational Science     Open Access   (Followers: 4)
Clinical Complementary Medicine and Pharmacology     Open Access  
Clinical Drug Investigation     Full-text available via subscription   (Followers: 7)
Clinical Medicine Insights : Therapeutics     Open Access  
Clinical Neuropharmacology     Hybrid Journal   (Followers: 2)
Clinical Pharmacist     Partially Free   (Followers: 12)
Clinical Pharmacokinetics     Full-text available via subscription   (Followers: 27)
Clinical Pharmacology & Therapeutics     Hybrid Journal   (Followers: 45)
Clinical Pharmacology in Drug Development     Hybrid Journal   (Followers: 4)
Clinical Pharmacology: Advances and Applications     Open Access   (Followers: 6)
Clinical Research and Regulatory Affairs     Hybrid Journal   (Followers: 9)
Clinical Therapeutics     Hybrid Journal   (Followers: 34)
Clinical Toxicology     Hybrid Journal   (Followers: 18)
Clinical Trials     Hybrid Journal   (Followers: 19)
CNS Drug Reviews     Open Access   (Followers: 4)
CNS Drugs     Full-text available via subscription   (Followers: 10)
Combination Products in Therapy     Open Access  
Consultant Pharmacist     Full-text available via subscription   (Followers: 2)
Consumer Drugs     Full-text available via subscription  
Contract Pharma     Full-text available via subscription  
Cosmetics     Open Access   (Followers: 4)
CPT : Pharmacometrics & Systems Pharmacology     Open Access   (Followers: 11)
Critical Reviews in Clinical Laboratory Sciences     Hybrid Journal   (Followers: 16)
Critical Reviews in Therapeutic Drug Carrier Systems     Full-text available via subscription   (Followers: 5)
Critical Reviews in Toxicology     Hybrid Journal   (Followers: 25)
Current Bioactive Compounds     Hybrid Journal  
Current Cancer Therapy Reviews     Hybrid Journal   (Followers: 5)
Current Clinical Pharmacology     Hybrid Journal   (Followers: 4)
Current Drug Delivery     Hybrid Journal   (Followers: 6)
Current Drug Discovery Technologies     Hybrid Journal   (Followers: 6)
Current Drug Metabolism     Hybrid Journal   (Followers: 5)
Current Drug Safety     Hybrid Journal   (Followers: 9)
Current Drug Targets     Hybrid Journal   (Followers: 4)
Current Drug Therapy     Hybrid Journal   (Followers: 3)
Current Enzyme Inhibition     Hybrid Journal   (Followers: 1)
Current Issues in Pharmacy and Medical Sciences     Open Access   (Followers: 2)
Current Medical Science     Hybrid Journal  
Current Medicinal Chemistry     Hybrid Journal   (Followers: 13)
Current Molecular Pharmacology     Hybrid Journal  
Current Nanoscience     Hybrid Journal  
Current Neuropharmacology     Hybrid Journal   (Followers: 1)
Current Opinion in Pharmacology     Hybrid Journal   (Followers: 9)
Current Pharmaceutical Analysis     Hybrid Journal   (Followers: 1)
Current Pharmaceutical Biotechnology     Hybrid Journal   (Followers: 10)
Current Pharmaceutical Design     Hybrid Journal   (Followers: 12)
Current Pharmacogenomics and Personalized Medicine     Hybrid Journal   (Followers: 3)
Current Pharmacology Reports     Hybrid Journal  
Current Protocols in Pharmacology     Hybrid Journal  
Current Radiopharmaceuticals     Hybrid Journal   (Followers: 1)
Current Research in Drug Discovery     Open Access   (Followers: 2)
Current Research in Pharmacology and Drug Discovery     Open Access   (Followers: 1)
Current Therapeutic Research     Open Access   (Followers: 6)
Current trends in Biotechnology and Pharmacy     Open Access   (Followers: 7)
Current Vascular Pharmacology     Hybrid Journal   (Followers: 4)
Dhaka University Journal of Pharmaceutical Sciences     Open Access  
Die Pharmazie - An International Journal of Pharmaceutical Sciences     Full-text available via subscription   (Followers: 5)
Dose-Response     Open Access  
Drug and Chemical Toxicology     Hybrid Journal   (Followers: 13)
Drug and Therapeutics Bulletin     Hybrid Journal   (Followers: 8)
Drug Delivery     Open Access   (Followers: 8)
Drug Delivery and Translational Research     Hybrid Journal   (Followers: 2)
Drug Design, Development and Therapy     Open Access   (Followers: 3)
Drug Development and Industrial Pharmacy     Hybrid Journal   (Followers: 30)
Drug Development Research     Hybrid Journal   (Followers: 11)
Drug Discovery Today: Technologies     Full-text available via subscription   (Followers: 12)
Drug Metabolism and Disposition     Hybrid Journal   (Followers: 11)
Drug Metabolism and Pharmacokinetics     Hybrid Journal   (Followers: 6)
Drug Metabolism Letters     Hybrid Journal   (Followers: 3)
Drug Metabolism Reviews     Hybrid Journal   (Followers: 8)
Drug Research     Hybrid Journal   (Followers: 3)
Drug Resistance Updates     Hybrid Journal   (Followers: 3)
Drug Safety     Full-text available via subscription   (Followers: 86)
Drug Safety - Case Reports     Open Access   (Followers: 2)
Drug Target Insights     Open Access  
Drug, Healthcare and Patient Safety     Open Access   (Followers: 10)
Drugs     Full-text available via subscription   (Followers: 152)
Drugs & Aging     Full-text available via subscription   (Followers: 10)
Drugs & Therapy Perspectives     Full-text available via subscription   (Followers: 9)
Drugs : Real World Outcomes     Hybrid Journal   (Followers: 1)
Drugs and Therapy Studies     Open Access  
Drugs in R & D     Full-text available via subscription   (Followers: 2)
Drugs of the Future     Full-text available via subscription   (Followers: 7)
East and Central African Journal of Pharmaceutical Sciences     Open Access   (Followers: 1)
Egyptian Pharmaceutical Journal     Open Access  
EJNMMI Radiopharmacy and Chemistry     Open Access  
EMC - Cosmetologia Medica e Medicina degli Inestetismi Cutanei     Full-text available via subscription  
Emerging Trends in Drugs, Addictions, and Health     Open Access   (Followers: 1)
Environmental Toxicology and Pharmacology     Hybrid Journal   (Followers: 9)
Epilepsy Research     Hybrid Journal   (Followers: 7)
Ethiopian Pharmaceutical Journal     Full-text available via subscription   (Followers: 1)
EUREKA : Health Sciences     Open Access  
European Journal of Clinical Pharmacology     Hybrid Journal   (Followers: 14)
European Journal of Drug Metabolism and Pharmacokinetics     Hybrid Journal   (Followers: 8)
European Journal of Hospital Pharmacy : Science and Practice (EJHP)     Hybrid Journal   (Followers: 5)
European Journal of Medicinal Plants     Open Access   (Followers: 2)
European Journal of Pharmaceutical Sciences     Hybrid Journal   (Followers: 89)
European Journal of Pharmaceutics and Biopharmaceutics     Hybrid Journal   (Followers: 35)
European Journal of Pharmacology     Hybrid Journal   (Followers: 8)
European Neuropsychopharmacology     Hybrid Journal   (Followers: 9)
European Review for Medical and Pharmacological Sciences     Full-text available via subscription   (Followers: 1)
Experimental and Clinical Psychopharmacology     Full-text available via subscription   (Followers: 8)

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Drugs
Journal Prestige (SJR): 1.547
Citation Impact (citeScore): 5
Number of Followers: 152  
 
  Full-text available via subscription Subscription journal
ISSN (Print) 0012-6667 - ISSN (Online) 1179-1950
Published by Adis Homepage  [21 journals]
  • Zavegepant: First Approval

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      Abstract: Zavegepant is a third generation, small-molecule, calcitonin gene-related peptide (CGRP) receptor antagonist being developed by Pfizer, under a license from Bristol-Myers Squibb, for the prevention and treatment of chronic and episodic migraine. In March 2023, zavegepant nasal spray (ZAVZPRET™) received its first approval in the USA for the acute treatment of migraine with or without aura in adults. Clinical development of an oral formulation of zavegepant is currently underway. This article summarizes the milestones in the development of zavegepant leading to this first approval for the acute treatment of migraine with or without aura in adults.
      PubDate: 2023-05-25
       
  • New Pharmacologic Approaches to the Treatment of Bipolar Depression

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      Abstract: Depression is the most commonly experienced mood state over the life span in individuals with bipolar disorder (BD) and is the primary driver of functional impairment and suicidality in BD. Despite this, there are few effective treatments for BD depression, with only a handful of atypical anti-psychotics and inconsistent evidence for traditional mood stabilizing agents. There have been few major ‘breakthroughs’ in the treatment of BD depression, and until recently, few agents that work via novel mechanisms of action to exert therapeutic effects. Here, we review treatments for BD depression which are emergent or on the horizon. Included are new atypical anti-psychotics, glutamate modulators (ketamine and cycloserine/lurasidone), neurosteroid modulators (zuranolone), anti-inflammatories and mitochondrial modulators, cannabidiol (CBD) and psilocybin. New atypical anti-psychotics lumateperone and cariprazine have demonstrated efficacy in large-scale, placebo-controlled, double-blind randomized controlled trials (RCT) in treatment of BD depression. Non-racemic amisulpride showed potential therapeutic benefit in one RCT which requires replication. Three small RCTs examined the efficacy of intravenous ketamine in BD depression and showed rapid antidepressant and anti-suicidal effects after a single infusion. Anti-inflammatory and mitochondrial modulators show inconsistent evidence for efficacy. There are currently no adequately powered RCTs of zuranolone, psilocybin or CBD in BD depression to support their use. While there are potentially efficacious, mechanistically novel agents on the horizon, they require further study and validation. Further investigation on how these agents may impact specific subgroups of patients will also advance the field.
      PubDate: 2023-05-25
       
  • Correction to: Eftrenonacog Alfa: A Review in Haemophilia B

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      PubDate: 2023-05-24
       
  • The Effect of Biological Treatment on Fatigue in
           Inflammatory Bowel Disease: A Systematic Review and Meta-analysis

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      Abstract: Background Fatigue is a frequent complaint in patients with inflammatory bowel disease. Biological drugs have demonstrated beneficial effects on some extraintestinal manifestations, but the effect on fatigue is not clear. Objective This study investigated the effects of biological and small molecule drugs approved for inflammatory bowel disease on fatigue. Methods We performed a systematic review and meta-analysis of randomized, placebo-controlled trials reporting Federal Drug Agency (FDA)-approved biological and small molecule drugs for use in ulcerative colitis and Crohn’s disease in which measures of fatigue were recorded before and after treatment. Only induction studies were included. Maintenance studies were excluded. We searched Embase (Ovid), Medline (Ovid), PsycINFO (Ovid), Cinahl (EBSCOhost), Web of Science Core Collection, Cochrane Central Register of Controlled Trials, and ClinicalTrials.gov in May 2022. Risk of bias was analyzed using the Cochrane risk-of-bias tool. Standardized mean difference was used to measure the treatment effect. Results A total of seven randomized controlled trials composed of 3835 patients were included in the meta-analysis. All of the studies included patients with moderately to severely active ulcerative colitis or Crohn’s disease. The studies used three different generic fatigue instruments: the Functional Assessment of Chronic Illness Therapy-Fatigue and the Short Form 36 Health Survey Vitality Subscale versions 1 and 2. Overall treatment with biological or small molecule agents showed a beneficial effect compared with placebo, with a standardized mean difference of 0.25 (95% confidence interval 0.15–0.34, p < 0.001). The effect was independent of type of drug or subtype of inflammatory bowel disease. Discussion The risk of bias was considered to be low for all domains except for missing outcome data. Even though the included studies were of high methodological quality, the review is limited by the small number of studies included and that the available studies were not designed to evaluate fatigue specifically. Conclusion Biological and small molecule drugs used in inflammatory bowel disease have a consistent, though small, beneficial effect on fatigue.
      PubDate: 2023-05-23
       
  • Rezafungin: First Approval

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      Abstract: Rezafungin (Rezzayo™), an intravenous once-weekly echinocandin that inhibits 1,3-β-d-glucan synthase, is being developed by Cidara Therapeutics. In March 2023, rezafungin received approval in the USA for the treatment of candidaemia and invasive candidiasis in patients aged ≥ 18 years who have limited or no alternative treatment options. Rezafungin is also being developed for the prevention of invasive fungal diseases in blood and marrow transplant recipients. This article summarizes the milestones in the development of rezafungin leading to the first approval for the treatment of candidaemia and invasive candidiasis.
      PubDate: 2023-05-22
       
  • Nociceptin Receptor-Related Agonists as Safe and Non-addictive Analgesics

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      Abstract: As clinical use of currently available opioid analgesics is often impeded by dose-limiting adverse effects, such as abuse liability and respiratory depression, new approaches have been pursued to develop safe, effective, and non-addictive pain medications. After the identification of the nociceptin/orphanin FQ (N/OFQ) peptide (NOP) receptor more than 25 years ago, NOP receptor-related agonists have emerged as a promising target for developing novel and effective opioids that modulate the analgesic and addictive properties of mu-opioid peptide (MOP) receptor agonists. In this review, we highlight the effects of the NOP receptor-related agonists compared with those of MOP receptor agonists in experimental rodent and more translational non-human primate (NHP) models and the development status of key NOP receptor-related agonists as potential safe and non-addictive analgesics. Several lines of evidence demonstrated that peptidic and non-peptidic NOP receptor agonists produce potent analgesic effects by intrathecal delivery in NHPs. Moreover, mixed NOP/MOP receptor partial agonists (e.g., BU08028, BU10038, and AT-121) display potent analgesic effects when administered intrathecally or systemically, without eliciting adverse effects, such as respiratory depression, itch behavior, and signs of abuse liability. More importantly, cebranopadol, a mixed NOP/opioid receptor agonist with full efficacy at NOP and MOP receptors, produces robust analgesic efficacy with reduced adverse effects, conferring promising outcomes in clinical studies. A balanced coactivation of NOP and MOP receptors is a strategy that warrants further exploration and refinement for the development of novel analgesics with a safer and effective profile. Graphical
      PubDate: 2023-05-20
       
  • Correction to: Recent Human Papillomavirus Vaccination is Associated with
           a Lower Risk of COVID-19: A US Database Cohort Study

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      PubDate: 2023-05-19
       
  • Results from Expanded Access Programs: A Review of Academic Literature

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      Abstract: Background Although expanded access is an increasingly used pathway for patients to access investigational medicine, little is known on the magnitude and content of published scientific research collected via expanded access. Methods We performed a review of all peer-reviewed expanded access publications between January 1, 2000 and January 1, 2022. We analyzed the publications for drugs, diseases, disease area, patient numbers, time, geographical location, subject, and research methodology (single center/multicenter, international/national, prospective/retrospective). We additionally analyzed endpoints reported in all COVID-19-related expanded access publications. Results We screened 3810 articles and included 1231, describing 523 drugs for 354 diseases for 507,481 patients. The number of publications significantly increased over time ( \(p<0.001\) ). Large geographical disparities existed as Europe and the Americas accounted for 87.4% of all publications, whereas Africa only accounted for 0.6%. Oncology and hematology accounted for 53% of all publications. Twenty-nine percent of all expanded access patients (N = 197,187) reported on in 2020 and 2021 were treated in the context of COVID-19. Conclusions By summarizing characteristics of patients, diseases, and research methods described in all scientific literature published on expanded access, we provide a unique dataset for future research. We show that published scientific research on expanded access has surged over the past decades, partly due to COVID-19. However, international collaboration and equity in geographic access remain an issue of concern. Lastly, we stress the need for harmonization of research legislation and guidance on the value of expanded access data within real-world data frameworks to improve equity in patient access and streamline future expanded access research.
      PubDate: 2023-05-18
       
  • Baricitinib for the Treatment of Alopecia Areata

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      Abstract: Alopecia areata (AA) is a relapsing, chronic, immune-mediated disease characterized by nonscarring, inflammatory hair loss that can affect any hair-bearing site. AA clinical presentation is heterogeneous. Its pathogenesis involves immune and genetic factors and several pro-inflammatory cytokines involved in AA pathogenesis, including interleukin-15 and interferon-γ, as well as Th2 cytokines, such as IL-4/IL-13, that signal through Janus kinase (JAK) pathway. AA treatment aims to stop its progression and reverse hair loss, and JAK inhibition has been shown to stop hair loss and reverse alopecia and has exhibited promising results in treating AA in clinical trials. Baricitinib, an oral, reversible, selective JAK1/JAK2 inhibitor, was shown to be superior to placebo on hair growth after 36 weeks of treatment in adults with severe AA in a phase 2 trial and recently in two phase 3 trials (BRAVE-AA1 and BRAVE-AA2). In both studies, the most common adverse events were upper respiratory tract infections, urinary tract infection, acne, headache, and elevated creatine kinase levels. On the basis of these trial results, baricitinib was recently approved by the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) for the treatment of adults with severe AA. Nevertheless, longer trials are needed to determine the long-term efficacy and safety of baricitinib in AA. Current trials are ongoing and are planned to remain randomized and blinded for up to 200 weeks.
      PubDate: 2023-05-17
       
  • Trofinetide: First Approval

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      Abstract: Trofinetide (DAYBUE™), an oral, small molecule, synthetic analog of glycine-proline-glutamate [GPE; the N-terminal tripeptide derivative of insulin like growth factor-1 (IGF-1)], is being developed by Neuren Pharmaceuticals and Acadia Pharmaceuticals for the treatment of rare childhood neurodevelopmental disorders. Trofinetide was approved in March 2023 in the USA for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older. This article summarizes the milestones in the development of trofinetide leading to this first approval for Rett syndrome.
      PubDate: 2023-05-16
       
  • Antibiotics and Probiotics for Irritable Bowel Syndrome

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      Abstract: Irritable bowel syndrome (IBS) is a disorder of a gut-brain interaction characterised by abdominal pain and a change in stool form or frequency. Current symptom-based definitions and the classification of IBS promote heterogeneity amongst patients, meaning that there may be several different pathophysiological abnormalities leading to similar symptoms. Although our understanding of IBS is incomplete, there are several indicators that the microbiome may be involved in a subset of patients. Techniques including a faecal sample analysis, colonic biopsies, duodenal aspirates or surrogate markers, such as breath testing, have been used to examine the gut microbiota in individuals with IBS. Because of a lack of a clear definition of what constitutes a healthy gut microbiota, and the fact that alterations in gut microbiota have only been shown to be associated with IBS, a causal relationship is yet to be established. We discuss several hypotheses as to how dysbiosis may be responsible for IBS symptoms, as well as potential treatment strategies. We review the current evidence for the use of antibiotics and probiotics to alter the microbiome in an attempt to improve IBS symptoms. Rifaximin, a non-absorbable antibiotic, is the most studied antibiotic and has now been licensed for use in IBS with diarrhoea in the USA, but the drug remains unavailable in many countries for this indication. Current evidence also suggests that certain probiotics, including Lactobacillus plantarum DSM 9843 and Bifidobacterium bifidum MIMBb75, may be efficacious in some patients with IBS. Finally, we describe the future challenges facing us in our attempt to modulate the microbiome to treat IBS.
      PubDate: 2023-05-15
       
  • Cipaglucosidase Alfa: First Approval

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      Abstract: Cipaglucosidase alfa (Pombiliti™) is a recombinant human acid α-glucosidase (GAA) product being developed by Amicus Therapeutics along with the enzyme stabilizer miglustat as a two-component therapy for Pompe disease. Pompe disease is a rare, inherited lysosomal disease caused by a deficiency of the enzyme GAA, which leads to accumulation of glycogen in various tissues. On 27 March 2023, cipaglucosidase alfa was approved in the EU as a long-term enzyme replacement therapy (ERT) used in combination with miglustat for the treatment of adults with late-onset Pompe disease. This article summarizes the milestones in the development of cipaglucosidase alfa leading to this first approval.
      PubDate: 2023-05-15
       
  • Retifanlimab: First Approval

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      Abstract: Retifanlimab (retifanlimab-dlwr; ZYNYZTM) is a programmed cell death 1 receptor-blocking antibody that is being developed by Incyte Corporation for the treatment of solid tumours, both as monotherapy and in combination with other agents. Retifanlimab recently received accelerated approval for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma. This article summarizes the milestones in the development of retifanlimab leading to this first approval for Merkel cell carcinoma.
      PubDate: 2023-05-15
       
  • Correction to: Comment on: ‘Should We Interfere with the Interleukin-6
           Receptor During COVID-19: What Do We Know'’

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      PubDate: 2023-05-11
       
  • Androgenetic Alopecia: Therapy Update

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      Abstract: Androgenetic alopecia (AGA), also known as male pattern hair loss (MPHL) or female pattern hair loss (FPHL), is the most common form of alopecia worldwide, and arises from an excessive response to androgens. AGA presents itself in a characteristic distribution unique to both sexes. Despite its prevalence, AGA can be quite challenging to treat. The condition is chronic in nature and stems from an interplay of genetic and environmental factors. There are only two US Food and Drug Administration (FDA)-approved drugs for the condition: topical minoxidil and oral finasteride. However, numerous non-FDA-approved treatments have been shown to be effective in treating AGA in various studies. Some of these treatments are relatively new and still to be explored, thus emphasizing the need for an updated review of the literature. In this comprehensive review, we discuss the evaluation of AGA and the mechanisms of action, costs, efficacies, and safety profiles of existing, alternative, and upcoming therapeutics for this widespread condition.
      PubDate: 2023-05-11
       
  • Ravulizumab: A Review in Generalised Myasthenia Gravis

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      Abstract: Ravulizumab (ULTOMIRIS®) is the first long-acting complement C5 inhibitor (administered intravenously every 8 weeks) to be approved in several countries globally, for adults with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody-positive (AChR Ab+). In the phase III CHAMPION MG trial, intravenous ravulizumab was associated with statistically significant improvements in the MG-Activities of Daily Living scale at week 26 of treatment compared with placebo in adults with AChR Ab+ gMG. Improvements in the Quantitative MG scale total score were also statistically significantly higher in ravulizumab than placebo recipients. These improvements were sustained to week 26 of treatment. Ravulizumab was generally well tolerated; the most common treatment-emergent adverse events were headache, diarrhoea and nausea. Efficacy and tolerability data for up to 1 year from the ongoing open-label extension phase are consistent with those from the randomized, placebo-controlled phase; further results are awaited with interest. Thus, ravulizumab is an efficacious, generally well tolerated and convenient treatment option in adults with AChR Ab+ gMG, expanding the options available for gMG management.
      PubDate: 2023-05-11
       
  • Omaveloxolone: First Approval

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      Abstract: Omaveloxolone (SKYCLARYS™) is an orally active, small molecule semi-synthetic triterpenoid drug that increases antioxidant activity, which is being developed by Reata Pharmaceuticals, Inc. for the treatment of Friedreich’s ataxia. In patients with Friedreich’s ataxia, the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway is suppressed, which is associated with oxidative stress, mitochondrial dysfunction and damage to cells, including central and peripheral neurones. The Nrf2 pathway may be activated by omaveloxolone as it blocks the ubiquitination and degradation of Nrf2. Omaveloxolone was approved in February 2023 in the USA for the treatment of Friedreich's ataxia. This article summarizes the milestones in the development of omaveloxolone leading to this first approval for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.
      PubDate: 2023-05-08
       
  • The Role of Vitamin D in Health and Disease: A Narrative Review on the
           Mechanisms Linking Vitamin D with Disease and the Effects of
           Supplementation

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      Abstract: Vitamin D insufficiency or deficiency (VDD) is a very prevalent condition in the general population. Vitamin D is necessary for optimal bone mineralization, but apart from the bone effects, preclinical and observational studies have suggested that vitamin D may have pleiotropic actions, whereas VDD has been linked to several diseases and higher all-cause mortality. Thus, supplementing vitamin D has been considered a safe and inexpensive approach to generate better health outcomes—and especially so in frail populations. Whereas it is generally accepted that prescribing of vitamin D in VDD subjects has demonstrable health benefits, most randomized clinical trials, although with design constraints, assessing the effects of vitamin D supplementation on a variety of diseases have failed to demonstrate any positive effects of vitamin D supplementation. In this narrative review, we first describe mechanisms through which vitamin D may exert an important role in the pathophysiology of the discussed disorder, and then provide studies that have addressed the impact of VDD and of vitamin D supplementation on each disorder, focusing especially on randomized clinical trials and meta-analyses. Despite there already being vast literature on the pleiotropic actions of vitamin D, future research approaches that consider and circumvent the inherent difficulties in studying the effects of vitamin D supplementation on health outcomes are needed to assess the potential beneficial effects of vitamin D. The evaluation of the whole vitamin D endocrine system, rather than only of 25-hydroxyvitamin D levels before and after treatment, use of adequate and physiologic vitamin D dosing, grouping based on the achieved vitamin D levels rather than the amount of vitamin D supplementation subjects may receive, and sufficiently long follow-up are some of the aspects that need to be carefully considered in future studies.
      PubDate: 2023-05-06
       
  • Authors’ Reply to Chia Siang Kow et al.: Comment on: ‘Should We
           Interfere with the Interleukin-6 Receptor During COVID-19: What Do We Know
           So Far'’

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      PubDate: 2023-05-04
       
  • Innovation in Targeted Intra-articular Therapies for Osteoarthritis

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      Abstract: Osteoarthritis is the most common chronic joint disease characterized by progressive damage to the joints, leading to pain and loss of function. There is currently no cure or disease-modifying therapy for osteoarthritis. Hence, the increasing disease prevalence linked with ageing and obesity represents a substantial socio-economic burden. Intra-articular therapy by injection of drugs into affected joints can optimize local drug bioavailability, while reducing risks of systemic toxicity, a concern in an ageing patient population. In this review, we investigate the current landscape of intra-articular drug therapies for osteoarthritis, including established approaches and those in clinical development. We performed a literature review using PubMed, complemented with a search for clinical trials using the ClinicalTrials.gov repository. Additionally, conference abstracts and presentations were identified and systematic snowballing was applied. Identified drugs were divided into several groups by main mechanism of action, and include drugs that reduce inflammation (anti-inflammatory), drugs aiming to prevent or reverse structural damage (structure modifying), drugs that aim to reduce the pain, and other drugs with a specific target. Most studies have been performed for osteoarthritis of the knee, a joint that is easily accessible for intra-articular treatments. Optimal therapy would provide symptomatic relief, while preventing further damage to the joint. The field of intra-articular drug therapies for osteoarthritis is rapidly evolving with clear challenges identified: definition of relevant outcome measures, optimization of clinical trial set-ups, and dealing with placebo responses. While many uncertainties persist, it appears that the innovation in drug development and improved clinical trial set-up may finally deliver successful therapies for this important disease.
      PubDate: 2023-04-17
       
 
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