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200 papers
Chronic Obstructive Pulmonary Disease (COPD): Developments in Pharmacological Treatments.
Singh D, Higham A, Mathioudakis AG … +1 more , Beech A
Drugs · 2025 Jul · PMID 40392521 · Full text
The immediate goals of pharmacological management in chronic obstructive pulmonary disease (COPD) are to minimise symptoms and improve exercise performance. The longer-term goals are to reduce the future risk of exacerba...
The immediate goals of pharmacological management in chronic obstructive pulmonary disease (COPD) are to minimise symptoms and improve exercise performance. The longer-term goals are to reduce the future risk of exacerbations, lung function decline and mortality. It is now recognised that a subset of COPD patients have type 2 inflammation, which is identified by the presence of higher blood eosinophil counts (BEC). Individuals with higher BEC show a greater response to pharmacological interventions targeting type 2 inflammation, including inhaled corticosteroids and the monoclonal antibody, dupilumab. The use of BEC as a biomarker to guide pharmacological treatment has enabled a precision medicine approach in COPD. This article reviews recent advances in the pharmacological treatment of COPD, encompassing the optimum use of inhaled combination treatments and the evidence to support the use of the novel inhaled phosphodiesterase inhibitor ensifentrine and monoclonal antibodies in patients with COPD.
Vonoprazan: A Review in Erosive Esophagitis and Non-Erosive Gastro-Esophageal Reflux Disease.
Fung S
Drugs · 2025 Jul · PMID 40388079 · Publisher ↗
Vonoprazan (Voquezna) is a potassium-competitive acid blocker (PCAB) with improved pharmacodynamic and pharmacokinetic properties compared with proton-pump inhibitors (PPIs) that allow for stronger control of gastric aci...
Vonoprazan (Voquezna) is a potassium-competitive acid blocker (PCAB) with improved pharmacodynamic and pharmacokinetic properties compared with proton-pump inhibitors (PPIs) that allow for stronger control of gastric acid with once-daily oral administration and no requirement to be taken in relation to timing of meals for optimal efficacy. In the USA, vonoprazan has been approved as a first-in-class treatment for healing and maintenance of healing of erosive esophagitis, and for relief of heartburn in adult patients with erosive esophagitis and non-erosive gastro-esophageal reflux disease (GERD). In pivotal phase III trials, vonoprazan was non-inferior to the PPI lansoprazole in healing and superior to lansoprazole in maintenance of healing of erosive esophagitis, and was superior to placebo for treating heartburn in US patients with non-erosive GERD. Vonoprazan was generally well tolerated; the most common adverse events included abdominal pain, constipation, diarrhea, nausea, and dyspepsia. Vonoprazan is, therefore, a valuable addition to the therapies available for adults with erosive esophagitis and non-erosive GERD.
Treatment of Advanced HIV in the Modern Era.
Garland JM, Mayan H, Kantor R
Drugs · 2025 Jul · PMID 40354016 · Full text
Antiretroviral therapy has transformed human immunodeficiency virus (HIV) infection from a fatal illness into a manageable chronic condition. However, despite remarkable progress, the HIV epidemic remains a global health...
Antiretroviral therapy has transformed human immunodeficiency virus (HIV) infection from a fatal illness into a manageable chronic condition. However, despite remarkable progress, the HIV epidemic remains a global health challenge, with ambitious targets such as 95-95-95 by 2030 at risk of being unmet. While antiretroviral therapy availability has expanded worldwide, gaps persist, including unawareness of HIV status, inconsistent medication uptake, and limited engagement in care across diverse settings. Advanced HIV represents a particularly challenging yet underexplored aspect of HIV care. Its definition is complex, complicating efforts to address the needs of this vulnerable population. This review characterizes advanced HIV populations, defines them by spectra of immune suppression, antiretroviral therapy exposure, and drug resistance, and explores contemporary approaches to their management, with a particular focus on drug resistance and its clinical implications in modern HIV care. It highlights the unique challenges faced by individuals presenting late to care, those with limited care engagement, and aging populations with long-term exposure to HIV and antiretroviral therapy. By defining these populations, refining our understanding of advanced HIV, and addressing the diverse needs of affected individuals, providers can enhance outcomes and develop strategies to overcome barriers to care. Bridging these critical gaps is essential to advancing global efforts to end the HIV epidemic, both in the USA and worldwide.
Navigating Psoriatic Arthritis: Treatment Pathways and Patient-Specific Strategies for Improved Outcomes.
Queiro R, Pinto-Tasende JA, Montilla-Morales C
Drugs · 2025 Jul · PMID 40350472 · Full text
Psoriatic arthritis (PsA) is a multifaceted chronic immune-mediated disease characterized by joint, skin, nail and entheseal involvement, affecting approximately 0.3-1% of the global population. In recent years, the trea...
Psoriatic arthritis (PsA) is a multifaceted chronic immune-mediated disease characterized by joint, skin, nail and entheseal involvement, affecting approximately 0.3-1% of the global population. In recent years, the treatment options for PsA have expanded from traditional nonsteroidal anti-inflammatory drugs and conventional synthetic disease-modifying antirheumatic drugs (DMARDs) to include biologic DMARDs and targeted synthetic DMARDs. Owing to the heterogeneity of the disease and prevalence of comorbidities, the selection and sequence of treatment are often unclear. In this narrative review, we outline the patient journey from diagnosis through various treatment lines, from conventional therapies to bDMARDS and tsDMARDs, and the considerations for treatment sequencing in patients who do not achieve an adequate response. We examine the factors influencing treatment response, such as disease severity, predominant disease domain, comorbidities, genetic variations, pharmacokinetic and immunogenicity issues. We highlight the importance of identifying robust biomarkers to predict response and the need to determine patient-specific factors, including the contribution of inflammatory mechanisms to disease activity, to inform treatment strategies and improve long-term outcomes. Promising results with more recently marketed biologic and targeted synthetic DMARDs, and the use of combination treatment approaches, offer new options for managing treatment-experienced patients.
The Role of CPX-351 in the Acute Myeloid Leukemia Treatment Landscape: Mechanism of Action, Efficacy, and Safety.
Pagano L, Danesi R, Benedetti E … +3 more , Morgagni R, Romani L, Venditti A
Drugs · 2025 Jul · PMID 40347359 · Full text
CPX-351 (also known as VYXEOS or Vyxeos liposomal) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 molar ratio and was the first example that utilized CombiPlex, a combination d...
CPX-351 (also known as VYXEOS or Vyxeos liposomal) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 molar ratio and was the first example that utilized CombiPlex, a combination drug technology platform. Superior efficacy with CPX-351 in the pivotal phase 3 randomized clinical trial versus its conventional free-drug counterpart led to its approval for the treatment of newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in multiple countries. Emerging evidence indicates that CPX-351 affords additional benefits compared with conventional chemotherapy, including protection against intestinal dysbiosis and fungal colonization, fewer infectious complications, and a lower incidence of cardiotoxicity. This review examines the mechanisms underlying CPX-351's therapeutic effects and highlights its expanding role in AML treatment by summarizing efficacy and safety data from preclinical models, the pivotal clinical trial, and real-world studies. Particular focus is given to recent findings on CPX-351's intestinal and cardioprotective properties, which together strengthen its safety and efficacy profile compared with conventional chemotherapy.
Lipid-Lowering Therapy and Cardiovascular Prevention in Elderly.
Baratta F, Moscucci F, Lospinuso I … +10 more , Cocomello N, Colantoni A, Di Costanzo A, Tramontano D, D'Erasmo L, Pastori D, Ettorre E, Del Ben M, Arca M, Desideri G
Drugs · 2025 Jun · PMID 40338434 · Full text
The global population aged 80 years and older will reach approximately half a billion in the coming years, and cardiovascular prevention in this group of patients will become a global health challenge. In the era of evid...
The global population aged 80 years and older will reach approximately half a billion in the coming years, and cardiovascular prevention in this group of patients will become a global health challenge. In the era of evidence-based medicine, the use of lipid-lowering therapies (LLTs) in the elderly, particularly in primary and secondary cardiovascular prevention, remains an area of active research. Although there is broad consensus on the use of LLTs in the elderly to prevent recurrent cardiovascular events in secondary prevention, there is considerable debate about their use in primary prevention. Many efforts have been made to improve cardiovascular risk stratification in patients over 75 years of age in primary prevention. In recent years, some specific risk scores have been developed, including the Systematic Coronary Risk Evaluation 2 for Older Persons (SCORE2-OP). While there are very few specific warnings to consider for LLTs in the elderly, an important challenge in this patient population is to identify the turning point at which the disutility risk outweighs the potential benefits. However, despite the widespread recognition of the importance of this issue, there is a lack of guidance on how to identify patients who should be withdrawn from therapy. The aim of this narrative review is to examine the current state of knowledge regarding the indications for LLT in elderly patients, identify outstanding issues, and discuss future developments.
Differentiating IL-23 Inhibitors in Crohn's Disease.
Nardone OM, Vuyyuru SK, Yuan Y … +2 more , Hanzel J, Jairath V
Drugs · 2025 Jun · PMID 40323342 · Publisher ↗
Interleukin-23 (IL-23), a member of the IL-12 family of cytokines, plays a critical role in intestinal homeostasis and inflammation and is strongly implicated in the pathogenesis of inflammatory bowel disease (IBD). Ther...
Interleukin-23 (IL-23), a member of the IL-12 family of cytokines, plays a critical role in intestinal homeostasis and inflammation and is strongly implicated in the pathogenesis of inflammatory bowel disease (IBD). Therapies targeting the p19 subunit of IL-23 have recently expanded the therapeutic options for IBD demonstrating efficacy and safety for the treatment of moderate to severe Crohn's disease (CD). Thus, in this review, we provide an overview of agents targeting the IL-23 pathway in CD, highlighting similarities and differences of specific IL-23 inhibitors. Furthermore, we summarize key phase 3 trials and head-to-head trials, focusing on design features and interpretation. Finally, we discuss the positioning of selective IL-23 agents for CD treatment along with areas of unmet clinical needs. However, real-world data will offer additional comparative effectiveness information, data for disease subtypes, and insights into the long-term outcomes of IL-23 inhibition. Looking ahead, ongoing phase 3 studies testing p19-specific selective IL-23 inhibitors are expected to expand the therapeutic options for patients with complex phenotypes, including those with extraintestinal manifestations (EIMs), fistulas, and strictures. Advances in molecular and cellular characterization, including the development of predictive molecular biomarkers, may help guide clinical decision-making, enabling more personalized treatment approaches. Precision medicine studies may further enhance our understanding of the molecular biology of IL-23, shedding light on how these agents work in complex CD and clarify their potential complementary or synergistic effects with other therapies.
Datopotamab Deruxtecan: First Approval.
Blair HA
Drugs · 2025 Jul · PMID 40323341 · Publisher ↗
Datopotamab deruxtecan (DATROWAY) is a trophoblast cell surface antigen 2 (TROP2)-directed antibody-drug conjugate being developed by Daiichi Sankyo and AstraZeneca for the treatment of solid tumours. On 27 December 2024...
Datopotamab deruxtecan (DATROWAY) is a trophoblast cell surface antigen 2 (TROP2)-directed antibody-drug conjugate being developed by Daiichi Sankyo and AstraZeneca for the treatment of solid tumours. On 27 December 2024, datopotamab deruxtecan was approved in Japan for the treatment of adult patients with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative unresectable or recurrent breast cancer after prior chemotherapy. The drug has since been approved on 17 January 2025 in the USA for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease. Datopotamab deruxtecan has also been approved in the EU for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative breast cancer who have received endocrine therapy and at least one line of chemotherapy in the advanced setting. Regulatory review of datopotamab deruxtecan in breast cancer is underway in Canada and China. This article summarizes the milestones in the development of datopotamab deruxtecan leading to this first approval for the treatment of HR positive, HER2 negative breast cancer.
Suzetrigine: First Approval.
Keam SJ
Drugs · 2025 Jun · PMID 40323340 · Publisher ↗
Suzetrigine (JOURNAVX™), an oral, non-opioid analgesic that is highly selective for the voltage-gated sodium channel NaV1.8 relative to other NaV channels, is being developed by Vertex Pharmaceuticals Inc. for the manage...
Suzetrigine (JOURNAVX™), an oral, non-opioid analgesic that is highly selective for the voltage-gated sodium channel NaV1.8 relative to other NaV channels, is being developed by Vertex Pharmaceuticals Inc. for the management of acute and neuropathic pain. NaV1.8 is expressed in peripheral pain-sensing neurons but is not expressed in the brain. Suzetrigine prevents transmission of pain signals by blocking NaV1.8 channels located along peripheral pain-sensing neurons, inhibiting the normal action potential. On 30 January 2025, suzetrigine was approved for the treatment of moderate to severe acute pain in adults in the USA. Suzetrigine is the first NaV inhibitor to be approved in this new therapeutic class of non-opioid analgesics. This article summarizes the milestones in the development of suzetrigine leading to this first approval for the treatment of moderate to severe acute pain.
Catumaxomab: First Approval.
Syed YY
Drugs · 2025 Jul · PMID 40304879 · Publisher ↗
Catumaxomab (Korjuny) is a first-in-class bispecific trifunctional rat-mouse hybrid monoclonal antibody currently under development with Lindis Biotech for malignant ascites, and bladder, gastric and ovarian cancers. It...
Catumaxomab (Korjuny) is a first-in-class bispecific trifunctional rat-mouse hybrid monoclonal antibody currently under development with Lindis Biotech for malignant ascites, and bladder, gastric and ovarian cancers. It binds epithelial cell adhesion molecule (EpCAM) on tumour cells and CD3 on T cells, while its Fc domain engages Fcγ receptor-positive accessory cells, bringing immune and tumour cells into close proximity to enhance tumour cell killing through multiple immunological mechanisms. Initially approved in the EU on 20 April 2009 for malignant ascites in adults with EpCAM+ carcinomas when standard therapy was unavailable or no longer feasible, catumaxomab was marketed by Fresenius Biotech GmbH (later Neovii Biotech GmbH) before being withdrawn on 2 June 2017 for commercial reasons. Lindis Biotech later acquired the rights and pursued reapproval. On 11 February 2025, catumaxomab was approved in the EU for the intraperitoneal treatment of malignant ascites in adults with EpCAM+ carcinomas who are not eligible for further systemic anticancer therapy. This article summarizes the milestones in the development of catumaxomab leading to this new approval.
Garadacimab: First Approval.
Fung S
Drugs · 2025 Jun · PMID 40261472 · Publisher ↗
Garadacimab (Andembry) is a fully human IgG4/lambda recombinant monoclonal anti-activated Factor XII antibody being developed by CSL Behring for the prevention of hereditary angioedema attacks. In January 2025, garadacim...
Garadacimab (Andembry) is a fully human IgG4/lambda recombinant monoclonal anti-activated Factor XII antibody being developed by CSL Behring for the prevention of hereditary angioedema attacks. In January 2025, garadacimab received its first approval in Australia and the UK for prevention of hereditary angioedema attacks in adult and adolescent patients aged ≥ 12 years. Additionally, in February 2025, garadacimab was approved for the same indication in the EU, Japan and Switzerland. In the USA and Canada, regulatory review of garadacimab is currently underway. This article summarizes the milestones in the development of garadacimab leading to this first approval for prevention of recurrent hereditary angioedema attacks in adult and adolescent patients aged ≥ 12 years.
Therapeutic Potential of Centhaquine Citrate, a Selective Alpha-2B Adrenoceptor Agonist, in the Management of Circulatory Shock.
Legrand M, Ranjan A, Rammohan S … +5 more , De Backer D, Ostermann M, Gulati A, Vincent JL, Khanna AK
Drugs · 2025 Jun · PMID 40253656 · Full text
Shock is a life-threatening condition marked by inadequate tissue perfusion and organ dysfunction with high morbidity and mortality. Activation of the sympatho-adrenergic system is pivotal in response to all four major c...
Shock is a life-threatening condition marked by inadequate tissue perfusion and organ dysfunction with high morbidity and mortality. Activation of the sympatho-adrenergic system is pivotal in response to all four major categories (i.e., hypovolemic, distributive, cardiogenic, and obstructive). In addition, exogenous vasopressors are often used to maintain organ perfusion pressure and decrease the size of the intravascular compartment. These agents preferentially constrict the arterial system but may lead to microcirculatory failure, especially at higher doses. This review outlines the sympatho-adrenergic system response after shock, discusses various vasopressors currently used as resuscitative agents, and reports the rationale for using a predominant venous vasopressor in shock. We also discuss the preliminary evidence for and ongoing research into a novel venous vasopressor, centhaquine citrate.
Crinecerfont: First Approval.
Lee A
Drugs · 2025 Jun · PMID 40240539 · Publisher ↗
Crinecerfont (CRENESSITY™) is a corticotropin releasing factor type 1 (CRF) receptor antagonist developed by Neurocrine Biosciences for the treatment of classic congenital adrenal hyperplasia (CAH) in adult and paediatri...
Crinecerfont (CRENESSITY™) is a corticotropin releasing factor type 1 (CRF) receptor antagonist developed by Neurocrine Biosciences for the treatment of classic congenital adrenal hyperplasia (CAH) in adult and paediatric patients. In patients with classic CAH, circulating levels of adrenocorticotropic hormone (ACTH), androstenedione and 17-hydroxyprogesterone are elevated, which traditionally has required supraphysiologic doses of glucocorticoids to manage. As a CRF receptor antagonist, crinecerfont acts by reducing systemic ACTH secretion to subsequently decrease elevated levels of steroid precursors and adrenal androgens, thereby reducing the dosage of glucocorticoids required to manage androgen levels in patients. This article summarizes the milestones in the development of crinecerfont leading to this first approval as an adjunctive treatment to glucocorticoid replacement to control androgens in adults and paediatric patients aged ≥ 4 years with classic CAH.
Association of Glucagon-Like Peptide-1 Receptor Agonist with Progression to Liver Cirrhosis and Alcohol-Related Admissions in Patients with Alcohol Use Disorder and Diabetes: A Retrospective Cohort Study.
Al-Moussally F, Khan S, Katukuri V … +2 more , Kinaan M, Mansi IA
Drugs · 2025 Jun · PMID 40223043 · Publisher ↗
AIM: In recent years, use of glucagon-like peptide-1 receptor agonists (GLP-1RA) has exponentially increased due to their beneficial effects on weight loss and cardiovascular outcomes. Lately, some animal studies and obs...
AIM: In recent years, use of glucagon-like peptide-1 receptor agonists (GLP-1RA) has exponentially increased due to their beneficial effects on weight loss and cardiovascular outcomes. Lately, some animal studies and observational data suggested that GLP-1RA may be useful in the treatment of alcohol use disorder (AUD). We aim to compare the risk of progression to liver cirrhosis and alcohol-related hospital admission after initiation of GLP-1RA versus dipeptidyl peptidase-4 inhibitors (DPP4i), as the active comparator, in patients with type 2 diabetes mellitus and AUD. METHODS: We conducted a retrospective propensity score-matched cohort study, utilizing new-user and active comparator design. The study used data from the Veterans Health Administration during fiscal years 2006 to 2021 encompassing adults with AUD who initiated either GLP-1RA or DPP4i prescriptions. Our two co-primary outcomes were progression to cirrhosis (compensated and decompensated cirrhosis) and alcohol-related hospital admission. RESULTS: The eligible cohort included 9965 GLP-1RA users and 19,688 DPP4i users. After propensity score matching, 7302 pairs were matched on 79 characteristics without residual imbalances. In the propensity score-matched cohort, progression to cirrhosis occurred in 6.6% of GLP-1RA users and 6.0% DPP4i users; odds ratio (OR): 1.1, 95% confidence interval (95% CI): 0.97-1.26. Alcohol-related hospital admission occurred in 1.4% of GLP-1RA users and in 1.7% of DPP4i users (OR: 0.85; 95% CI: 0.65-1.11). CONCLUSIONS: Use of GLP-1RA in patients with AUD was not associated with beneficial effect on progression to cirrhosis or alcohol-related hospital admission.
Correction: Current and Emerging Therapies for Lysosomal Storage Disorders.
Abelleyra Lastoria DA, Keynes S, Hughes D
Drugs · 2025 Jun · PMID 40220089 · Publisher ↗
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PI3K Inhibitors as Potential Therapeutic Agents for the Treatment of COPD with Associated Atherosclerosis.
Cazzola M, Page C, Rogliani P … +2 more , Calzetta L, Matera MG
Drugs · 2025 Jun · PMID 40214902 · Full text
Chronic obstructive pulmonary disease (COPD) and cardiovascular disease (CVD) share a complex and multifactorial relationship characterized by overlapping risk factors, systemic inflammation, and intertwined pathophysiol...
Chronic obstructive pulmonary disease (COPD) and cardiovascular disease (CVD) share a complex and multifactorial relationship characterized by overlapping risk factors, systemic inflammation, and intertwined pathophysiological mechanisms, with atherosclerosis emerging as a central inflammatory process connecting COPD and CVD, driven by systemic inflammation, oxidative stress, and endothelial dysfunction. While systemic inflammation is recognized as a critical link between these conditions, the precise pathways through which inflammation arises remain under investigation. There is therefore a need for therapeutic strategies to mitigate cardiovascular risks in patients with COPD. Among the pathways contributing to this interplay, the phosphoinositide 3-kinase (PI3K) signaling pathway has gained significant attention. Dysregulated PI3K signaling contributes to inflammation, oxidative stress, and endothelial dysfunction, which are key drivers of both COPD and CVD. Consequently, PI3K inhibitors have emerged as a promising therapeutic approach to mitigate inflammation and oxidative damage, offering a targeted strategy to address the shared pathological mechanisms underlying these diseases. A comprehensive understanding of the role of PI3K signaling and its inhibitors could facilitate the development of novel interventions to reduce cardiovascular risk in patients with COPD.
A systematic review of macro-, meso, and micro-level harm reduction interventions addressing the U.S. opioid overdose epidemic.
McCormick KA, Samora J, Claborn KR … +2 more , Steiker LKH, DiNitto DM
Drugs (Abingdon Engl) · 2025 · PMID 40206199 · Full text
BACKGROUND: This systematic review aimed to 1) identify the range of opioid harm reduction interventions implemented at macro-, meso-, and micro-levels in the United States, and 2) summarize the outcomes of these interve...
BACKGROUND: This systematic review aimed to 1) identify the range of opioid harm reduction interventions implemented at macro-, meso-, and micro-levels in the United States, and 2) summarize the outcomes of these interventions. METHODS: We conducted a systematic review of academic literature published between 2011-2023 following PRISMA guidelines. Articles were excluded if they reported on research that was not specific to opioids, did not report the effects of an intervention, or focused on a medical treatment for opioid use disorder. Two coders independently extracted data and reconciled discrepancies prior to narrative synthesis. RESULTS: Of 6,198 articles initially identified, 36 met inclusion criteria across macro (=7), meso (=8), and micro (=21) domains. Positive evidence for micro- and meso-level interventions is largely consistent, whereas evidence for macro-level interventions is mixed. Among micro- and meso-level interventions, supply distribution interventions were most effective in increasing safe use knowledge and behaviors among people who use drugs. DISCUSSION: Most harm reduction interventions demonstrate moderate to strong evidence of effectiveness for addressing the opioid overdose epidemic across domains. Findings revealed a lack of multi-level interventions and a lack of culturally relevant interventions that prioritize Black and Brown communities disproportionately impacted during the opioid overdose epidemic's latter phases.
Acoramidis: First Approval.
Lee A
Drugs · 2025 Jun · PMID 40180779 · Publisher ↗
Disassociation of transthyretin (TTR) has been identified as a key step in the pathology of TTR amyloid cardiomyopathy, which is characterised by the presence of amyloid fibrils in cardiac tissue. Acoramidis (ATTRUBY™) i...
Disassociation of transthyretin (TTR) has been identified as a key step in the pathology of TTR amyloid cardiomyopathy, which is characterised by the presence of amyloid fibrils in cardiac tissue. Acoramidis (ATTRUBY™) is a small molecule TTR stabiliser being developed by BridgeBio Pharma, Inc. and is the first drug to demonstrate near-complete (> 90%) stabilisation of TTR. This article summarizes the milestones in the development of acoramidis leading to its first approval in the USA for the treatment of the cardiomyopathy of wild-type or variant TTR-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization. In the EU, a positive opinion has been adopted for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy.
The Potential of RNA Therapeutics in Treating Cardiovascular Disease.
Mainkar G, Ghiringhelli M, Zangi L
Drugs · 2025 May · PMID 40175855 · Publisher ↗
Despite significant advances in cardiology over the past few decades, cardiovascular diseases (CVDs) remain the leading cause of global mortality and morbidity. This underscores the need for novel therapeutic interventio...
Despite significant advances in cardiology over the past few decades, cardiovascular diseases (CVDs) remain the leading cause of global mortality and morbidity. This underscores the need for novel therapeutic interventions that go beyond symptom management to address the underlying causal mechanisms of CVDs. RNA-based therapeutics represent a new class of drugs capable of regulating specific genetic and molecular pathways, positioning them as strong candidates for targeting the root causes of a wide range of diseases. Moreover, owing to the vast diversity in RNA form and function, these molecules can be utilized to induce changes at different levels of gene expression regulation, making them suitable for a broad array of medical applications, even within a single disease context. Several RNA-based therapies are currently being investigated for their potential to address various CVD pathologies. These include treatments aimed at promoting cardiac revascularization and regeneration, preventing cardiomyocyte apoptosis, reducing harmful circulating cholesterols and fats, lowering blood pressure, reversing cardiac fibrosis and remodeling, and correcting the genetic basis of inherited CVDs. In this review, we discuss the current landscape of RNA therapeutics for CVDs, with an emphasis on their classifications, modes of action, advancements in delivery strategies and considerations for their implementation, as well as CVD targets with proven therapeutic potential.
Race/Ethnicity and Contexts: Associations of Racial/Ethnic Discrimination with Underage Youth's Alcohol Use and Drinking Contexts.
Lipperman-Kreda S, Antin TMJ, Wharton K … +1 more , Wilson I
Drugs (Abingdon Engl) · 2025 · PMID 40170950 · Full text
BACKGROUND: This study examined how racial/ethnic discrimination may shape alcohol use behaviors, related problems, and contexts of alcohol use among underage youth (<21 years old). Age group differences were also explor...
BACKGROUND: This study examined how racial/ethnic discrimination may shape alcohol use behaviors, related problems, and contexts of alcohol use among underage youth (<21 years old). Age group differences were also explored. METHODS: Cross-sectional data were obtained from California youth identified as racial and ethnic minoritized youth (n=605; ages 14-20). An adapted version of the Everyday Discrimination Scale was used to assess racial/ethnic discrimination. Demographics were also assessed. Outcomes included drinking status, past-6-month alcohol frequency and quantity, number of types of alcohol-related problems, risk for alcohol addiction, and past-6-month alcohol frequency and quantity in public and private settings. Multinomial, negative binomial, and linear regression analyses were conducted, controlling for demographics and exposure to context settings. RESULTS: Experiencing racial/ethnic discrimination was positively associated with lifetime drinking, any past-6-month drinking, and risk for alcohol addiction. It was also positively associated with the past-6-month typical number of drinks participants consumed in public settings. Age-group differences were observed with some greater risks for older underage youth (18-20 years old). CONCLUSIONS: Findings suggest the importance of trauma-informed interventions for youth of diverse racial/ethnic groups and for addressing racial/ethnic discrimination in schools or communities to address alcohol use inequities in young people.
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