keyword
https://read.qxmd.com/read/37891814/pharmacological-therapies-of-spinal-muscular-atrophy-a-narrative-review-of-preclinical-clinical-experimental-and-real-world-evidence
#21
REVIEW
Salvatore Crisafulli, Brigida Boccanegra, Giacomo Vitturi, Gianluca Trifirò, Annamaria De Luca
Spinal muscular atrophy (SMA) is a rare neuromuscular disease, with an estimated incidence of about 1 in 10,000 live births. To date, three orphan drugs have been approved for the treatment of SMA: nusinersen, onasemnogene abeparvovec, and risdiplam. The aim of this narrative review was to provide an overview of the pre- and post-marketing evidence on the pharmacological treatments approved for the treatment of SMA by identifying preclinical and clinical studies registered in clinicaltrials.gov and in the EU PAS register from their inception until the 4 January 2023...
October 10, 2023: Brain Sciences
https://read.qxmd.com/read/37890129/elexacaftor-tezacaftor-ivacaftor-treatment-and-depression-related-events
#22
JOURNAL ARTICLE
Bonnie Ramsey, Christoph U Correll, David R DeMaso, Edward McKone, Elizabeth Tullis, Jennifer L Taylor-Cousar, Chenghao Chu, Nataliya Volkova, Neil Ahluwalia, David Waltz, Simon Tian, Marcus A Mall
RATIONALE: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (pwCF) aged 2 years and older with ≥1 F508del-CFTR allele. Following US approval in 2019, reports emerged of depression-related adverse events in pwCF treated with ELX/TEZ/IVA. OBJECTIVES: To review available evidence on depression-related events in pwCF treated with ELX/TEZ/IVA in context of background epidemiology in pwCF. METHODS: Safety data from 14 ELX/TEZ/IVA clinical trials and 10 trials of CFTR modulators in which placebo was administered, along with data from CF registries in the US and Germany and cumulative post-marketing adverse event data from 61,499 pwCF who initiated ELX/TEZ/IVA following initial approval in the US (October 2019) through October 2022, were reviewed and used to calculate exposure-adjusted rates of depression-related adverse events and prevalence of depression...
October 27, 2023: American Journal of Respiratory and Critical Care Medicine
https://read.qxmd.com/read/37882379/the-introduction-of-the-cgrp-monoclonal-antibodies-and-their-effect-on-the-prescription-patterns-of-chronic-migraine-preventive-medications-in-a-tertiary-headache-center-a-retrospective-observational-analysis
#23
JOURNAL ARTICLE
Leon S Moskatel, Anna Graber-Naidich, Zihuai He, Niushen Zhang
OBJECTIVE: To determine the effect of the introduction of the calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) in 2018 on the prescribing of older medications for the prevention of chronic migraine. BACKGROUND: Prior to 2018, the preventive treatment of migraine borrowed from medications intended to treat other illnesses with the last medication, onabotulinumtoxinA, receiving Food and Drug Administration (FDA) approval for the prevention of chronic migraine in 2010...
October 26, 2023: Headache
https://read.qxmd.com/read/37877189/prehevbrio-the-first-approved-3-antigen-hepatitis-b-vaccine
#24
REVIEW
Timo Vesikari, Joanne M Langley, Vlad Popovic, Francisco Diaz-Mitoma
INTRODUCTION: Hepatitis B remains a major cause of death and morbidity worldwide. Universal childhood immunization programs have been very successful, but many adults remain unprotected or are not optimally protected. PreHevbrio [Hepatitis B Vaccine (recombinant)]is a highly immunogenic 3-antigen (S/pre-S1/pre-S2) hepatitis B vaccine (3A-HBV) that recently received marketing authorization in the United States (2021), the European Union, United Kingdom (2022 - brand name PreHevbri), and Canada (2022- brand name PreHevbrio) for the prevention of infection caused by all known subtypes of the hepatitis B virus and the delta virus in adults 18 years and older...
October 25, 2023: Expert Review of Vaccines
https://read.qxmd.com/read/37875437/-technical-guidelines-for-seasonal-influenza-vaccination-in-china-2023-2024
#25
JOURNAL ARTICLE
(no author information available yet)
Influenza is an acute respiratory infectious disease that is caused by the influenza virus, which seriously affects human health. The influenza virus has frequent antigenic drifts that can facilitate escape from pre-existing population immunity and lead to the rapid spread and annual seasonal epidemics. Influenza outbreaks occur in crowded settings, such as schools, kindergartens, and nursing homes. Seasonal influenza epidemics can cause 3-5 million severe cases and 290 000-650 000 respiratory disease-related deaths worldwide every year...
October 10, 2023: Zhonghua Liu Xing Bing Xue za Zhi, Zhonghua Liuxingbingxue Zazhi
https://read.qxmd.com/read/37864395/differences-in-time-to-patient-access-to-innovative-cancer-medicines-in-six-european-countries
#26
JOURNAL ARTICLE
Julie M Vancoppenolle, Nora Franzen, Simone N Koole, Valesca P Retèl, Wim H van Harten
Patients across Europe face inequity regarding access to anticancer medicines. While access is typically evaluated through reimbursement status or sales data, patients can receive first access through early access programs (EAPs) or off-label use. This study aims to assess the time to patient access at the hospital level, considering different indications and countries. (Pre-)registered access to six innovative medicines (Olaparib, Niraparib, Ipilimumab, Osimeritinib, Nivolumab and Ibritunib) was measured using a cross-sectional survey...
October 20, 2023: International Journal of Cancer. Journal International du Cancer
https://read.qxmd.com/read/37836465/eight-weeks-of-daily-cannabidiol-supplementation-improves-sleep-quality-and-immune-cell-cytotoxicity
#27
JOURNAL ARTICLE
Jacob N Kisiolek, Victoria A Flores, Arjun Ramani, Blake Butler, James M Haughian, Laura K Stewart
BACKGROUND: The endocannabinoid system is active in nervous and immune cells and involves the expression of two cannabinoid receptor genes (CB1 and CB2), along with endogenous endocannabinoid ligands, 2-arachidonoyl glycerol (2-AG) and arachidonoyl ethanolamide (anandamide), and their synthetic enzymes. Cannabidiol (CBD) is a non-intoxicating exogenous cannabinoid agonist derived from plants that, at high doses, has received FDA approval as an anticonvulsant for epileptic seizures, and at low doses is marketed as a food-grade supplement for improved mental health, sleep quality, and immunological function...
September 27, 2023: Nutrients
https://read.qxmd.com/read/37829441/tolerability-of-subcutaneous-ofatumumab-with-long-term-exposure-in-relapsing-multiple-sclerosis
#28
JOURNAL ARTICLE
John Kramer, Ralf Linker, David Paling, Adam Czaplinski, Olaf Hoffmann, V Wee Yong, Noreen Barker, Amy Perrin Ross, Elisabeth Lucassen, Mohammad Gufran, Xixi Hu, Ronald Zielman, Gustavo Seifer, Patrick Vermersch
BACKGROUND: Ofatumumab is approved for treating relapsing multiple sclerosis (RMS). Examining tolerability will enable understanding of its risk-benefit profile. OBJECTIVE: Report the tolerability profile of ofatumumab in RMS during treatment of up to 4 years and the effect of pre-medication. METHODS: Cumulative data from the overall safety population included patients taking continuous ofatumumab or being newly switched from teriflunomide...
2023: Multiple Sclerosis Journal—Experimental, Translational and Clinical
https://read.qxmd.com/read/37797226/eyes-on-the-prize-decoding-the-ophthalmic-product-regulations-and-intricacies-of-the-u-s-food-and-drug-administration-approval
#29
JOURNAL ARTICLE
Khemraj Hirani, Mylarrao Bansinath, Rahul Mittal, Joana R N Lemos, Emily Adis, Priyanka Poojari, Jane Margaret Igoe, Marcio R Soares, Sanjoy Bhattacharya, Roy E Weiss
The dynamic and continuously evolving field of ophthalmology necessitates rigorous regulatory oversight in the United States. This review outlines the multifaceted Food and Drug Administration's (FDA) approval process for ophthalmic products, detailing the classifications, pathways, and regulatory compliance for devices, drugs, biologics, and combination products. Particular emphasis is placed on distinct frameworks for Class I, II, and III devices, as well as regulations for drugs, biologics, and combination products...
October 5, 2023: Journal of Ocular Pharmacology and Therapeutics
https://read.qxmd.com/read/37766194/covid-19-vaccines-over-three-years-after-the-outbreak-of-the-covid-19-epidemic
#30
REVIEW
Aleksandra Anna Zasada, Aniela Darlińska, Aldona Wiatrzyk, Katarzyna Woźnica, Kamila Formińska, Urszula Czajka, Małgorzata Główka, Klaudia Lis, Paulina Górska
The outbreak of COVID-19 started in December 2019 and spread rapidly all over the world. It became clear that the development of an effective vaccine was the only way to stop the pandemic. It was the first time in the history of infectious diseases that the process of the development of a new vaccine was conducted on such a large scale and accelerated so rapidly. At the end of 2020, the first COVID-19 vaccines were approved for marketing. At the end of March 2023, over three years after the outbreak of the COVID-19 pandemic, 199 vaccines were in pre-clinical development and 183 in clinical development...
August 23, 2023: Viruses
https://read.qxmd.com/read/37765160/repurposable-drugs-for-immunotherapy-and-strategies-to-find-candidate-drugs
#31
REVIEW
Norihiro Sakai, Kenya Kamimura, Shuji Terai
Conventional drug discovery involves significant steps, time, and expenses; therefore, novel methods for drug discovery remain unmet, particularly for patients with intractable diseases. For this purpose, the drug repurposing method has been recently used to search for new therapeutic agents. Repurposed drugs are mostly previously approved drugs, which were carefully tested for their efficacy for other diseases and had their safety for the human body confirmed following careful pre-clinical trials, clinical trials, and post-marketing surveillance...
August 24, 2023: Pharmaceutics
https://read.qxmd.com/read/37729719/harnessing-autologous-immune-effector-mechanisms-in-acute-myeloid-leukemia-2023-update-of-trials-and-tribulations
#32
REVIEW
Shyam A Patel, Elisa Bello, Andrew Wilks, Jonathan M Gerber, Narayanan Sadagopan, Jan Cerny
Numerous recent advances have been made in therapeutic approaches toward acute myeloid leukemia (AML). Since 2017, we have seen eleven novel Food & Drug Administration (FDA)-approved medications for AML, all of which extend beyond the classical cytarabine-based cytostatic chemotherapy. In the recent two decades, the role of immune surveillance in AML has been intensively investigated. The power of one's own innate and adaptive immunity has been harnessed pharmacologically toward the goal of clearance of AML cells...
November 2023: Leukemia Research
https://read.qxmd.com/read/37701158/procedural-sedation-in-a-uae-emergency-department-encouraging-informed-decision-making-through-a-patient-information-leaflet
#33
JOURNAL ARTICLE
Amani Khamis AlBedwawi, Afra Bakheet Almansoori, Muna Abdelaziz Aljasmi, Fatema Salem Al Ameri, Nasser Ahmed, Abdul-Salam Adnan Al Mnaseer, Ismail Mohamed Al Ramahi, Kenneth Charles Dittrich, Hasan Qayyum
Introduction Procedural sedation is a common procedure conducted in emergency departments (ED) across the world, which requires patients to receive anesthesia/sedation medication in a controlled environment in order to alleviate pain, anxiety, and suffering, thereby allowing multiple procedures to be completed in a safe and timely manner. We deploy this technique for joint reductions, burns dressings, wound repairs, etc. in our ED. As a large tertiary referral hospital ED, we aimed to benchmark our practice for this high-acuity procedure against international standards...
September 2023: Curēus
https://read.qxmd.com/read/37686024/using-cfdna-and-ctdna-as-oncologic-markers-a-path-to-clinical-validation
#34
REVIEW
Jonathan Dao, Patrick J Conway, Baskaran Subramani, Devi Meyyappan, Sammy Russell, Daruka Mahadevan
The detection of circulating tumor DNA (ctDNA) in liquid biopsy samples as an oncological marker is being used in clinical trials at every step of clinical management. As ctDNA-based liquid biopsy kits are developed and used in clinics, companies work towards increased convenience, accuracy, and cost over solid biopsies and other oncological markers. The technology used to differentiate ctDNA and cell-free DNA (cfDNA) continues to improve with new tests and methodologies being able to detect down to mutant allele frequencies of 0...
August 25, 2023: International Journal of Molecular Sciences
https://read.qxmd.com/read/37680352/comparability-strategy-and-demonstration-for-post-approval-production-cell-line-change-of-a-bevacizumab-biosimilar-ibi305
#35
JOURNAL ARTICLE
Zhouyi Wu, Gangling Xu, Wu He, Chuanfei Yu, Wanqiu Huang, Shirui Zheng, Dian Kang, Michael H Xie, Xingjun Cao, Lan Wang, Kaikun Wei
High-producing cell line could improve the affordability and availability of biotherapeutic products. A post-approval production cell line change, low-titer CHO-K1S to high-titer CHO-K1SV GS-KO, was performed for a China marketed bevacizumab biosimilar IBI305. Currently, there is no regulatory guideline specifically addressing the requirements for comparability study of post-approval cell line change, which is generally regarded as the most complex process change for biological products. Following the quality by design principle and risk assessment, an extensive analytical characterization and three-way comparison was performed by using a panel of advanced analytical methods...
July 2023: Antibody Therapeutics
https://read.qxmd.com/read/37670318/out-of-balance-conflicts-of-interest-persist-in-food-chemicals-determined-to-be-generally-recognized-as-safe
#36
JOURNAL ARTICLE
Klara Matouskova, Thomas G Neltner, Maricel V Maffini
Manufacturers of chemicals added to food are responsible for determining that the use of their products is safe. There are two major legal definitions of chemicals in food: (1) food additives which includes ingredients and chemicals indirectly entering food from packaging and processing equipment, and (2) generally recognized as safe (GRAS) substances mostly used as ingredients. The law requires food additives to undergo approval by the U.S. Food and Drug Administration (FDA) before they are sold, but it GRAS substances are exempted from pre-market approval...
September 6, 2023: Environmental Health
https://read.qxmd.com/read/37659896/developmental-and-reproductive-safety-of-advax-cpg55-2%C3%A2-adjuvanted-covid-19-and-influenza-vaccines-in-mice
#37
JOURNAL ARTICLE
Isaac G Sakala, Yoshikazu Honda-Okubo, Nikolai Petrovsky
SpikoGen® is a recombinant spike protein vaccine against COVID-19 that obtained marketing authorization in the Middle East on October 6th , 2021, becoming the first adjuvanted protein-based COVID-19 vaccine of its type to achieve approval. SpikoGen® vaccine utilizes a unique adjuvant Advax-CpG55.2, which comprises delta inulin and CpG55.2 oligonucleotide, a synthetic human toll-like receptor (TLR)-9 agonist. As part of a safety assessment, developmental and reproductive toxicity (DART) studies were undertaken in mice of Advax-CpG55...
August 31, 2023: Vaccine
https://read.qxmd.com/read/37630999/parenteral-ready-to-use-fixed-dose-combinations-including-nsaids-with-paracetamol-or-metamizole-for-multimodal-analgesia-approved-products-and-challenges
#38
REVIEW
Fernando Silva, Gustavo Costa, Francisco Veiga, Catarina Cardoso, Ana Cláudia Paiva-Santos
The combination of non-steroidal anti-inflammatory drugs (NSAIDs) with non-opioid analgesics is common in clinical practice for the treatment of acute painful conditions like post-operative and post-traumatic pain. Despite the satisfactory results achieved by oral analgesics, parenteral analgesia remains a key tool in the treatment of painful conditions when the enteral routes of administration are inconvenient. Parenteral ready-to-use fixed-dose combinations of non-opioid analgesics combinations, including NSAIDs and paracetamol or metamizole, could play a central role in the treatment of painful conditions by combining the advantages of multimodal and parenteral analgesia in a single formulation...
July 31, 2023: Pharmaceuticals
https://read.qxmd.com/read/37592154/identification-of-confusing-medicine-proprietary-names-toward-safe-medicine-use-a-cross-sectional-study-in-sri-lanka
#39
JOURNAL ARTICLE
Manori Jayasinghe, Thotawaththage Loshadhi Indunika Srilal, Sewwandi Subasinghe, Shukry Zawahir
BACKGROUND: Look-alike sound-alike (LASA) medications have similar pronunciation (phonetic) and/or manifestation (orthographic), which could create confusion among users and challenge the safe use of medicines. The availability of foreign products in local markets aggravates the situation. This study was designed to examine the registered medicine proprietary names in Sri Lanka to discern the presence of similar medicine names in the industry. METHODS: A cross-sectional study was conducted on the registered drug proprietary names in Sri Lanka...
August 17, 2023: Therapeutic Innovation & Regulatory Science
https://read.qxmd.com/read/37546576/challenges-and-opportunities-for-incentivising-antibiotic-research-and-development-in-europe
#40
REVIEW
Michael Anderson, Dimitra Panteli, Robin van Kessel, Gunnar Ljungqvist, Francesca Colombo, Elias Mossialos
Antimicrobial, and particularly antibiotic resistance are one of the world's biggest challenges today, and urgent action is needed to reinvigorate the antibiotic development pipeline. To inform policy discussions during and after the 2023 Swedish Presidency of the Council of the European Union, we critically appraise incentive options recently proposed by the European Commission, and member states, and consider what has been achieved over the last two decades in relation to antibiotic research and development...
October 2023: The Lancet regional health. Europe
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