journal
https://read.qxmd.com/read/38592603/relugolix-estradiol-norethisterone-acetate-a-review-in-endometriosis-associated-pain
#1
JOURNAL ARTICLE
Hannah A Blair
An oral fixed-dose combination of relugolix/estradiol/norethisterone (also known as norethindrone) acetate [Myfembree® (USA); Ryeqo® (EU)] (hereafter referred to as relugolix combination therapy) has been approved in the USA for the management of moderate to severe pain associated with endometriosis in premenopausal women and in the EU for the symptomatic treatment of endometriosis in adult women of reproductive age with a history of previous medical or surgical treatment for their endometriosis. The gonadotropin-releasing hormone (GnRH) receptor antagonist relugolix decreases estradiol and progesterone levels, while the addition of estradiol/norethisterone acetate mitigates hypoestrogenic effects including bone mineral density (BMD) loss and vasomotor symptoms...
April 9, 2024: Drugs
https://read.qxmd.com/read/38573485/the-pharmacological-landscape-for-fatty-change-of-the-pancreas
#2
JOURNAL ARTICLE
Maxim S Petrov
The quest for medications to reduce intra-pancreatic fat deposition is now quarter a century old. While no specific medication has been approved for the treatment of fatty change of the pancreas, drug repurposing shows promise in reducing the burden of the most common disorder of the pancreas. This leading article outlines the 12 classes of medications that have been investigated to date with a view to reducing intra-pancreatic fat deposition. Information is presented hierarchically-from preclinical studies to retrospective findings in humans to prospective interventional studies to randomised controlled trials...
April 4, 2024: Drugs
https://read.qxmd.com/read/38554243/perfluorohexyloctane-ophthalmic-solution-a-review-in-dry-eye-disease
#3
REVIEW
Amy Zhuang-Yan, Yahiya Y Syed
Perfluorohexyloctane ophthalmic solution (Miebo® ) is a single-entity, water-, steroid- and preservative-free, first-in-class semifluorinated alkane that is approved in the USA for the treatment of the signs and symptoms of dry eye disease (DED). DED is often linked with meibomian gland dysfunction (MGD), which causes an excessive evaporation of tears. Perfluorohexyloctane ophthalmic solution stabilizes the lipid layer of the tear film and inhibits tear evaporation by forming a monolayer at the air-liquid interface...
March 30, 2024: Drugs
https://read.qxmd.com/read/38546957/correction-eplontersen-first-approval
#4
Tina Nie
No abstract text is available yet for this article.
March 28, 2024: Drugs
https://read.qxmd.com/read/38546956/dimdazenil-first-approval
#5
REVIEW
Yahiya Y Syed
Dimdazenil (Junoenil® ) is a small-molecule, oral, partial positive allosteric modulator of the gamma-aminobutyric acid (GABA)A receptor that is being developed by Zhejiang Jingxin Pharmaceutical in collaboration with Evotec for the treatment of insomnia. Dimdazenil is designed to overcome issues associated with full GABAA receptor agonists, such as tolerance, withdrawal symptoms and associated adverse effects. On 29 November 2023, dimdazenil oral capsules received approval in China for the short-term treatment of insomnia...
March 28, 2024: Drugs
https://read.qxmd.com/read/38528311/correction-aponermin-first-approval
#6
Sohita Dhillon
No abstract text is available yet for this article.
March 26, 2024: Drugs
https://read.qxmd.com/read/38528310/danicopan-first-approval
#7
REVIEW
Connie Kang
Danicopan (Voydeya® ) is an oral complement factor D inhibitor that is being developed by Alexion AstraZeneca Rare Disease as add-on treatment to ravulizumab or eculizumab for patients with clinically significant extravascular haemolysis. Danicopan recently received approval in Japan for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) when used in addition to a complement component 5 (C5) inhibitor. Subsequently, the European Medicines Agency adopted a positive opinion recommending the granting of marketing authorisation for danicopan for the treatment of patients with PNH who continue to have residual haemolytic anaemia despite treatment with a complement C5 inhibitor...
March 26, 2024: Drugs
https://read.qxmd.com/read/38517653/iptacopan-first-approval
#8
REVIEW
Yahiya Y Syed
Iptacopan (FABHALTA® ) is an oral complement Factor B inhibitor developed by Novartis Pharmaceuticals for the treatment of complement-mediated diseases. Acting upstream of complement 5 in the alternative pathway, iptacopan inhibits both terminal complement-mediated intravascular haemolysis and complement 3-mediated extravascular haemolysis. On 5 December 2023, iptacopan received approval in the USA for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). This article summarizes the milestones in the development of iptacopan leading to this first approval for PNH...
March 22, 2024: Drugs
https://read.qxmd.com/read/38499937/correction-zilucoplan-first-approval
#9
Matt Shirley
No abstract text is available yet for this article.
March 19, 2024: Drugs
https://read.qxmd.com/read/38489088/correction-lumasiran-a-review-in-primary-hyperoxaluria-type-1
#10
Connie Kang
No abstract text is available yet for this article.
March 15, 2024: Drugs
https://read.qxmd.com/read/38480630/drugs-targeting-cd20-in-multiple-sclerosis-pharmacology-efficacy-safety-and-tolerability
#11
REVIEW
Alise K Carlson, Moein Amin, Jeffrey A Cohen
Currently, there are four monoclonal antibodies (mAbs) that target the cluster of differentiation (CD) 20 receptor available to treat multiple sclerosis (MS): rituximab, ocrelizumab, ofatumumab, and ublituximab. B-cell depletion therapy has changed the therapeutic landscape of MS through robust efficacy on clinical manifestations and MRI lesion activity, and the currently available anti-CD20 mAb therapies for use in MS are a cornerstone of highly effective disease-modifying treatment. Ocrelizumab is currently the only therapy with regulatory approval for primary progressive MS...
March 14, 2024: Drugs
https://read.qxmd.com/read/38480629/tamoxifen-dose-de-escalation-an-effective-strategy-for-reducing-adverse-effects
#12
REVIEW
Sanne M Buijs, Stijn L W Koolen, Ron H J Mathijssen, Agnes Jager
Tamoxifen, a cornerstone in the adjuvant treatment of estrogen receptor-positive breast cancer, significantly reduces breast cancer recurrence and breast cancer mortality; however, its standard adjuvant dose of 20 mg daily presents challenges due to a broad spectrum of adverse effects, contributing to high discontinuation rates. Dose reductions of tamoxifen might be an option to reduce treatment-related toxicity, but large randomized controlled trials investigating the tolerability and, more importantly, efficacy of low-dose tamoxifen in the adjuvant setting are lacking...
March 14, 2024: Drugs
https://read.qxmd.com/read/38478331/comparative-efficacy-of-drug-interventions-on-nafld-over-24-weeks-a-traditional-and-network-meta-analysis-of-randomized-controlled-trials
#13
Yifan Wang, He Yi, Weixia Sun, Hekai Yu, Wenxuan Tao, Xiaojin Yu, Dianrong Jia, Yingzhao Liu, Stephen J Pandol, Ling Li
BACKGROUND AND AIMS: Nonalcoholic fatty liver disease (NAFLD), currently referred to as metabolic dysfunction-associated steatotic liver disease (MASLD), affects approximately 38% of the world's population, yet no pharmacological therapies have been approved for treatment. We conducted a traditional and network meta-analysis to comprehensively assess the effectiveness of drug regimens on NAFLD, and continued to use the old terminology for consistency. METHODS: Randomized, placebo-controlled trials (RCTs) investigating drug therapy in an adult population diagnosed with NAFLD with or without diabetes mellitus were included...
March 13, 2024: Drugs
https://read.qxmd.com/read/38472707/fidanacogene-elaparvovec-first-approval
#14
REVIEW
Sohita Dhillon
Fidanacogene elaparvovec (Pr BEQVEZ™) is an adeno-associated viral (AAV) vector-based gene therapy developed by Spark Therapeutics (a subsidiary of Roche) and Pfizer (under a license from Spark Therapeutics) for the treatment of haemophilia B. In December 2023, fidanacogene elaparvovec received its first approval for the treatment of adults (aged ≥ 18 years) with moderately severe to severe haemophilia B (congenital factor IX deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74 (AAVRh74var)...
March 12, 2024: Drugs
https://read.qxmd.com/read/38451443/opioids-for-acute-musculoskeletal-pain-a-systematic-review-with-meta-analysis
#15
Caitlin M P Jones, Aili Langford, Chris G Maher, Christina Abdel Shaheed, Richard Day, Chung-Wei Christine Lin
OBJECTIVE: To evaluate the efficacy of opioids for people with acute musculoskeletal pain against placebo. STUDY DESIGN: Systematic review and meta-analyses of randomised, placebo-controlled trials of opioid analgesics for acute musculoskeletal pain in any setting. The primary outcomes were pain and disability at the immediate timepoint (< 24 h). DATA SOURCES: Multiple databases were searched from their inception to February 22nd, 2023...
March 7, 2024: Drugs
https://read.qxmd.com/read/38441807/adult-onset-still-s-disease-aosd-advances-in-understanding-pathophysiology-genetics-and-emerging-treatment-options
#16
REVIEW
Sara Bindoli, Chiara Baggio, Andrea Doria, Paolo Sfriso
Adult-onset Still's disease (AOSD) is a multisystemic complex disorder clinically characterised by episodes of spiking fever, evanescent rash, polyarthritis or diffuse arthralgias; multiorgan involvement may develop according to the hyper-inflammatory extent. The pathogenesis of AOSD is not completely recognised. The central role of macrophage activation, which results in T helper 1 (Th1) cell cytokine activation, is well established. Pro-inflammatory cytokines such as interleukin (IL)-1, IL-6 and IL-18 play a fundamental role in disease onset and progression...
March 5, 2024: Drugs
https://read.qxmd.com/read/38441806/ser-109-vowst-%C3%A2-a-review-in-the-prevention-of-recurrent-clostridioides-difficile-infection
#17
REVIEW
Hannah A Blair
SER-109 (VOWST™ ; fecal microbiota spores, live-brpk) is a live biotherapeutic product indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in patients 18 years of age and older following standard of care (SOC) antibacterial treatment for recurrent CDI. It is a purified bacterial spore suspension sourced from healthy donors. As the first oral faecal microbiota product approved for prevention of recurrent CDI, SER-109 is administered as four capsules once daily for three consecutive days...
March 5, 2024: Drugs
https://read.qxmd.com/read/38441805/aponermin-first-approval
#18
REVIEW
Sohita Dhillon
Aponermin () is a recombinant circularly permuted human tumour necrosis factor-related apoptosis-inducing ligand (TRAIL) developed by Beijing Sunbio Biotech (a wholly owned subsidiary of Wuhan Hiteck Biological Pharma CO., LTD) for the treatment of multiple myeloma. Aponermin binds to and activates the death receptors 4 and/or 5 on tumour cells, triggering intracellular caspase reactions and inducing apoptosis, thereby exerting antitumor effects. In November 2023, aponermin in combination with thalidomide and dexamethasone received its first approval in China for the treatment of patients with relapsed or refractory multiple myeloma who have received at least two prior therapies...
March 5, 2024: Drugs
https://read.qxmd.com/read/38418773/microencapsulated-benzoyl-peroxide-for-rosacea-in-context-a-review-of-the-current-treatment-landscape
#19
REVIEW
Seemal R Desai, Hilary Baldwin, James Q Del Rosso, Richard L Gallo, Neal Bhatia, Julie C Harper, Jean Philippe York, Linda Stein Gold
Rosacea, a chronic skin condition affecting millions of people in the USA, leads to significant social and professional stigmatization. Effective management strategies are crucial to alleviate symptoms and improve patients' quality of life. Encapsulated benzoyl peroxide 5% (E-BPO 5%) is a newly FDA-approved topical treatment for rosacea that shows promise in enhancing therapeutic response and minimizing skin irritation. This review aims to assess the role of recently FDA approved E-BPO 5% in the current treatment landscape for rosacea management, as it is not yet included in clinical guidelines that predominantly rely on older approved therapies...
February 29, 2024: Drugs
https://read.qxmd.com/read/38418772/apadamtase-alfa-first-approval
#20
REVIEW
Young-A Heo
Apadamtase alfa (ADAMTS13, recombinant-krhn; ADZYNMA), a human recombinant form of a disintegrin and metalloproteinase with thrombospondin motifs 13 (ADAMTS13), is being developed by Takeda under license from KM biologics for thrombotic thrombocytopenic purpura (TTP) and sickle cell disease. On 9 November 2023, apadamtase alfa was approved in the USA for prophylactic and on-demand enzyme replacement therapy (ERT) in paediatric and adult patients with congenital TTP. Apadamtase alfa is under regulatory review for congenital TTP in the EU and Japan, and is under clinical development for immune-mediated TTP in several countries worldwide...
February 29, 2024: Drugs
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