First Gene Therapy Approved in U.S.
Title: First Gene Therapy Approved in U.S.
Category: Health News
Created: 8/30/2017 12:00:00 AM
Last Editorial Review: 8/31/2017 12:00:00 AM
Title: First Gene Therapy Approved in U.S.
Category: Health News
Created: 8/30/2017 12:00:00 AM
Last Editorial Review: 8/31/2017 12:00:00 AM
Title: Goats Can Read Human Emotions, Prefer Happy Faces
Category: Health News
Created: 8/31/2018 12:00:00 AM
Last Editorial Review: 8/31/2018 12:00:00 AM
Title: Nourianz Approved to Treat 'Off' Episodes in Parkinson Disease
Category: Health News
Created: 8/28/2019 12:00:00 AM
Last Editorial Review: 8/29/2019 12:00:00 AM
Title: Scientists Challenge Key Survival Stat Cited by U.S. Officials in Plasma Approval
Category: Health News
Created: 8/25/2020 12:00:00 AM
Last Editorial Review: 8/25/2020 12:00:00 AM
Title: FDA Approves Wider Use of Plasma as Coronavirus Treatment
Category: Health News
Created: 8/24/2020 12:00:00 AM
Last Editorial Review: 8/25/2020 12:00:00 AM
Title: FDA Approves New Rapid Coronavirus Test
Category: Health News
Created: 8/27/2020 12:00:00 AM
Last Editorial Review: 8/27/2020 12:00:00 AM
Title: Rapid 15-Minute COVID-19 Test Approved By FDA
Category: Health News
Created: 8/27/2020 12:00:00 AM
Last Editorial Review: 8/28/2020 12:00:00 AM
Title: Moderna Readies for Full Vaccine Approval, as Pfizer Submits Data on Booster Shot
Category: Health News
Created: 8/26/2021 12:00:00 AM
Last Editorial Review: 8/26/2021 12:00:00 AM
Title: Don't Forget to Apply Sunscreen Before & After Water Fun
Category: Health News
Created: 8/27/2021 12:00:00 AM
Last Editorial Review: 8/27/2021 12:00:00 AM
Title: Pfizer Asks FDA to Approve Omicron-Specific Booster Shot
Category: Health News
Created: 8/22/2022 12:00:00 AM
Last Editorial Review: 8/23/2022 12:00:00 AM
Title: Many Seniors Love Pickleball, But Injuries Can Happen
Category: Health News
Created: 8/6/2022 12:00:00 AM
Last Editorial Review: 8/8/2022 12:00:00 AM
Title: 'Stepped' Approach to Treating Diabetic Eye Disease May Be Best
Category: Health News
Created: 7/15/2022 12:00:00 AM
Last Editorial Review: 7/15/2022 12:00:00 AM
Title: What Happens If We Get AIDS?
Category: Diseases and Conditions
Created: 6/10/2022 12:00:00 AM
Last Editorial Review: 6/10/2022 12:00:00 AM
Title: New Approach Cuts Odds for Anal Cancer in People With HIV
Category: Health News
Created: 6/16/2022 12:00:00 AM
Last Editorial Review: 6/16/2022 12:00:00 AM
High-throughput sequencing (HTS) technologies have been instrumental in investigating biological questions at the bulk and single-cell levels. Comparative analysis of two HTS data sets often relies on testing the statistical significance for the difference of two negative binomial distributions (DOTNB). Although negative binomial distributions are well studied, the theoretical results for DOTNB remain largely unexplored. Here, we derive basic analytical results for DOTNB and examine its asymptotic properties. As a state-of-the-art application of DOTNB, we introduce DEGage, a computational method for detecting differentially expressed genes (DEGs) in scRNA-seq data. DEGage calculates the mean of the sample-wise differences of gene expression levels as the test statistic and determines significant differential expression by computing the P-value with DOTNB. Extensive validation using simulated and real scRNA-seq data sets demonstrates that DEGage outperforms five popular DEG analysis tools: DEGseq2, DEsingle, edgeR, Monocle3, and scDD. DEGage is robust against high dropout levels and exhibits superior sensitivity when applied to balanced and imbalanced data sets, even with small sample sizes. We utilize DEGage to analyze prostate cancer scRNA-seq data sets and identify marker genes for 17 cell types. Furthermore, we apply DEGage to scRNA-seq data sets of mouse neurons with and without fear memory and reveal eight potential memory-related genes overlooked in previous analyses. The theoretical results and supporting software for DOTNB can be widely applied to comparative analyses of dispersed count data in HTS and broad research questions.
ABSTRACTTraining health workers is one of the biggest challenges and cost drivers when introducing a new contraceptive method or service delivery innovation. PATH developed a digital training curriculum for family planning providers who are learning to offer subcutaneous DMPA (DMPA-SC), including through self-injection, as an option among a range of contraceptive methods. The DMPA-SC eLearning course for health workers includes 10 lessons with an emphasis on informed choice counseling and training clients to self-inject. In partnership with Ministries of Health in Senegal and Uganda, the course was rolled out in select areas in 2019–2020, including during the COVID-19 pandemic when physical distancing requirements restricted in-person training. We conducted evaluations in both countries to assess the practical application of this digital training approach for contraceptive introduction. The evaluation consisted of a post-training survey, an observational assessment conducted during post-training supportive supervision, and an estimation of training costs.In both countries, a majority (88.6% in Uganda and 64.3% in Senegal) scored above 80% on a DMPA-SC knowledge test following the training. In Senegal, where there was a comparison group of providers trained in person, those providers scored similar on the post-test to eLearners. Providers in both groups and in both countries felt more prepared to administer DMPA-SC or offer self-injection to clients after receiving a supervision visit (93%–98% of eLearners felt very prepared after supervision as compared to 45%–72% prior). The evaluation results suggest that digital approaches offer a number of benefits, can be cost-effective, and are most optimal when blended with in-person training and/or supportive supervision.
The diagnostic work-up of patients with fever of unknown origin (FUO) begins with a thorough history and physical examination, complete blood count with differential, chest x-ray, urinalysis and culture, electrolyte panel, liver enzymes, erythrocyte sedimentation rate, and C-reactive protein level. Additional imaging procedures, including nuclear medicine tests, are generally used as second-line procedures, with 18F-FDG PET and PET/CT assuming increasingly important roles in the diagnostic work-up. The Society of Nuclear Medicine and Molecular Imaging, the Infectious Diseases Society of America, and the American College of Nuclear Medicine convened an autonomous expert work group to comprehensively review the published literature for nuclear imaging in adults and children with FUO and establish appropriate use criteria (AUC). This process was performed in accordance with the Protecting Access to Medicare Act of 2014, which requires that all referring physicians consult AUC by using a clinical decision support mechanism before ordering advanced diagnostic imaging services. The complete findings and discussions of the work group were published on January 8, 2023, and are available at https://www.snmmi.org/ClinicalPractice/content.aspx?ItemNumber=15666. The AUC in the final document are intended to assist referring health care providers in appropriate use of nuclear medicine imaging procedures in patients with FUO. The work group noted limitations in the current literature on nuclear medicine imaging for FUO, with the need for well-designed prospective multicenter investigations. Consensus findings from published data and expert opinions were used to create recommendations in common clinical scenarios for adults and children. Included in the complete document is a discussion of inflammation of unknown origin (IUO), a recently described entity. In view of the fact that the criteria for FUO and IUO are similar (except for fever > 38.3°C [100.9°F]) and that the most common etiologies of these 2 entities are similar, it is the expert opinion of the work group that the recommendations for nuclear medicine imaging of FUO are also applicable to IUO. These recommendations are included in the full guidance document. This summary reviews rationale, methodology, and main findings and refers the reader to the complete AUC document.
[177Lu]Lu-PSMA-617 was approved by the U.S. Food and Drug Administration for patients with prostate-specific membrane antigen (PSMA)–positive metastatic castration-resistant prostate cancer (mCRPC). Since the time of regulatory approval, however, real-world data have been lacking. This study investigated the efficacy, safety, and outcome predictors of [177Lu]Lu-PSMA-617 at a major U.S. academic center. Methods: Patients with mCRPC who received [177Lu]Lu-PSMA-617 at the Johns Hopkins Hospital outside clinical trials were screened for inclusion. Patients who underwent [177Lu]Lu-PSMA-617 and had available outcome data were included in this study. Outcome data included prostate-specific antigen (PSA) response (≥50% decline), PSA progression-free survival (PFS), and overall survival (OS). Toxicity data were evaluated according to the Common Terminology Criteria for Adverse Events version 5.03. The study tested the association of baseline circulating tumor DNA mutational status in homologous recombination repair, PI3K alteration pathway, and aggressive-variant prostate cancer–associated genes with treatment outcome. Baseline PSMA PET/CT images were analyzed using SelectPSMA, an artificial intelligence algorithm, to predict treatment outcome. Associations with the observed treatment outcome were evaluated. Results: All 76 patients with PSMA-positive mCRPC who received [177Lu]Lu-PSMA-617 met the inclusion criteria. A PSA response was achieved in 30 of 74 (41%) patients. The median PSA PFS was 4.1 mo (95% CI, 2.0–6.2 mo), and the median OS was 13.7 mo (95% CI, 11.3–16.1 mo). Anemia of grade 3 or greater, thrombocytopenia, and neutropenia were observed in 9 (12%), 3 (4%), and 1 (1%), respectively, of 76 patients. Transient xerostomia was observed in 23 (28%) patients. The presence of aggressive-variant prostate cancer–associated genes was associated with a shorter PSA PFS (median, 1.3 vs. 6.3 mo; P = 0.040). No other associations were observed between circulating tumor DNA mutational status and treatment outcomes. Eighteen of 71 (25%) patients classified by SelectPSMA as nonresponders had significantly lower rates of PSA response than patients classified as likely responders (6% vs. 51%; P < 0.001), a shorter PSA PFS (median, 1.3 vs. 6.3 mo; P < 0.001), and a shorter OS (median, 6.3 vs. 14.5 mo; P = 0.046). Conclusion: [177Lu]Lu-PSMA-617 offered in a real-world setting after regulatory approval in the United States demonstrated antitumor activity and a favorable toxicity profile. Artificial-intelligence–based analysis of baseline PSMA PET/CT images may improve patient selection. Validation of these findings on larger cohorts is warranted.
This review explores the concept of synergy in pharmacology, emphasizing its importance in optimizing treatment outcomes through the combination of drugs with different mechanisms of action. Synergy, defined as an effect greater than the expected additive effect elicited by individual agents according to specific predictive models, offers a promising approach to enhance therapeutic efficacy while minimizing adverse events. The historical evolution of synergy research, from ancient civilizations to modern pharmacology, highlights the ongoing quest to understand and harness synergistic interactions. Key concepts, such as concentration-response curves, additive effects, and predictive models, are discussed in detail, emphasizing the need for accurate assessment methods throughout translational drug development. Although various mathematical models exist for synergy analysis, selecting the appropriate model and software tools remains a challenge, necessitating careful consideration of experimental design and data interpretation. Furthermore, this review addresses practical considerations in synergy assessment, including preclinical and clinical approaches, mechanism of action, and statistical analysis. Optimizing synergy requires attention to concentration/dose ratios, target site localization, and timing of drug administration, ensuring that the benefits of combination therapy detected bench-side are translatable into clinical practice. Overall, the review advocates for a systematic approach to synergy assessment, incorporating robust statistical analysis, effective and simplified predictive models, and collaborative efforts across pivotal sectors, such as academic institutions, pharmaceutical companies, and regulatory agencies. By overcoming critical challenges and maximizing therapeutic potential, effective synergy assessment in drug development holds promise for advancing patient care.
Combining drugs with different mechanisms of action for synergistic interactions optimizes treatment efficacy and safety. Accurate interpretation of synergy requires the identification of the expected additive effect. Despite innovative models to predict the additive effect, consensus in drug-drug interactions research is lacking, hindering the bench-to-bedside development of combination therapies. Collaboration among science, industry, and regulation is crucial for advancing combination therapy development, ensuring rigorous application of predictive models in clinical settings.
Hearing disorders pose significant challenges to individuals experiencing them and their overall quality of life, emphasizing the critical need for advanced pharmacological approaches to address these conditions. Current treatment options often focus on amplification devices, cochlear implants, or other rehabilitative therapies, leaving a substantial gap regarding effective pharmacological interventions. Advancements in our understanding of the molecular and cellular mechanisms involved in hearing disorders induced by noise, aging, and ototoxicity have opened new avenues for drug development, some of which have led to numerous clinical trials, with promising results. The development of optimal drug delivery solutions in animals and humans can also enhance the targeted delivery of medications to the ear. Moreover, large genome studies contributing to a genetic understanding of hearing loss in humans combined with advanced molecular technologies in animal studies have shown a great potential to increase our understanding of the etiologies of hearing loss. The auditory system exhibits circadian rhythms and temporal variations in its physiology, its vulnerability to auditory insults, and its responsiveness to drug treatments. The cochlear clock rhythms are under the control of the glucocorticoid system, and preclinical evidence suggests that the risk/benefit profile of hearing disorder treatments using chronopharmacological approaches would be beneficial. If translatable to the bedside, such approaches may improve the outcome of clinical trials. Ongoing research into the molecular and genetic basis of auditory disorders, coupled with advancements in drug formulation and delivery as well as optimized timing of drug administration, holds great promise of more effective treatments.
Hearing disorders pose significant challenges to individuals and their overall quality of life, emphasizing the critical need for advanced pharmacological approaches to address these conditions. Ongoing research into the molecular and genetic basis of auditory disorders, coupled with advancements in drug delivery procedures and optimized timing of drug administration, holds the promise of more effective treatments.
Metastatic porocarcinomas (PCs) are vanishingly rare, highly aggressive skin adnexal tumors with mortality rates exceeding 70%. Their rarity has precluded the understanding of their disease pathogenesis, let alone the conduct of clinical trials to evaluate treatment strategies. There are no effective agents for unresectable PCs. Here, we successfully demonstrate how functional precision medicine was implemented in the clinic for a metastatic PC with no known systemic treatment options. Comprehensive genomic profiling of the tumor specimen did not yield any actionable genomic aberrations. However, ex vivo drug testing predicted pazopanib efficacy, and indeed, administration of pazopanib elicited remarkable clinicoradiological response. Pazopanib and its class of drugs should be evaluated for efficacy in other cases of PC, and the rationale for efficacy should be determined when PC tumor models become available. A functional precision medicine approach could be useful to derive effective treatment options for rare cancers.
The deadline to claim part of the $15 million Cash App settlement is November 18. Here's what to know.
It will likely release in 2026 and feature Apple Intelligence, according to a reliable analyst.
Two runners saved by the same public defibrillator appeal for all the life-saving devices to be publicly available. Tens of thousands of defibs are unregistered so ‘invisible’ in an emergency.
Chris suffers from "The Curse of the Steam Deck." Kelley can't pickpocket, so she mugs people at night. And Ryan likes practical grave robbers. Curl up in your clothmap quilt and enjoy our cozy '60s demon world.
The post RPG Cast – Episode 681: “Ganon Is Afraid of Homemade Apple Pie” appeared first on RPGamer.
We’re still a week away from Blizzard’s Warcraft 30th Anniversary Direct next Wednesday the 13th of November, but art from an apparent remaster of 1995 real time strategy game Warcraft II: Tides Of Darkness has leaked online, via Xibbly user Stiven. It’s a thin one, as far as leaks go, but does show what looks to be cover, logo art, and a Battle.net icon. Thanks for the spot, Percy Coswald Gamer.
GeForce Now, Nvidia’s PC-focused game streaming service, will begin calling time on its most muscular of power users. A post on the GFN subreddit announced the introduction of a 100-hour monthly cap (or "allowance"), effective from January 1st 2025 for anyone who signs up after that date. Existing streamists, or anyone who signs up by the end of 2024, will get a year’s grace period before the limit kicks in from January 2026.
After nearly a year of public beta honing, the Nvidia App – Team Green’s new one-stop shop for desktop GPU management – is out in full. Not alongside the upcoming RTX 50 series, as rumoured, but right-now-today-this-minute. I’ve been testing out the launch version and while it’s not without some dud features, it does agreeably achieve its stated goal of combining the functions within Nvidia Control Panel and GeForce Experience. And if installing it means never having to use the latter again, well, that’s 149MB well spent.
“It might feel like 2064 on the surface, but in its nostalgic, rechargeable heart, the show parties like it’s 1999.”
Comet Nishimura (C/2023 P1) was discovered in August and is now whizzing by Earth, but finding it in the sky will be a challenge.
More than 80 per cent of B.C.'s water basins are experiencing level 4 or 5 drought conditions, with salmon in many parts of the province struggling to make it to their spawning grounds.
President-elect Donald Trump on Tuesday announced that Tesla founder Elon Musk and businessman Vivek Ramaswamy will a new Department of Government Efficiency, a White House office given the task of cutting the federal budget.
Apple recently announced its new lineup of Macs and rolled out Apple Intelligence, its latest artificial intelligence-powered feature for its products.
The Portuguese midfielder is settling into life in east London well after his loan move.
If rumours are true and this one should be, I started it, we have a special edition of the Weekend show where we talk about the evolution of the role of the CIO with two incredible CIOs as the CIO Association of Canada turns 20. Don’t miss it. MUSIC UP Can HP make you love […]
The post Hashtag Trending Mar.1- HP debacle; Humanoid robots closer to hitting our workplaces; Apple blew $10 billion on the electric car before pulling the plug first appeared on ITBusiness.ca.Apple Music gives a whole new meaning to the phrase the hits just keep on coming. It’s not the opposing candidates, it’s public AI systems that are spreading election disinformation, and LockBit, the cybercriminal gang may be back from the dead and saying so long to the ChatGPT plugins, which went from innovation to legacy […]
The post Hashtag Trending Mar.5- Apple Music fined for market dominance; LockBit back from the dead; OpenAI kills ChatGPT plugins first appeared on ITBusiness.ca.Using a laser-based detection system, archaeologists have discovered over 6,500 pre-Hispanic structures -- including a previously unknown Maya city named Valeriana -- in Campeche, Mexico.
The post Laser Mapping Reveals Previously Unknown Maya City with Stone Pyramids in Mexico appeared first on Sci.News: Breaking Science News.
Speculation has already run rampant on who Florida Gov. Ron DeSantis will appoint to replace Sen. Marco Rubio if Rubio becomes President-elect Trump's secretary of state.
William McGinley is returning to the Trump White House to serve as his White House Counsel, President-elect Trump announced.
Social media commentators claimed Jill Biden refused to look at Vice President Harris as they were seated together at Arlington Cemetery for a Veterans' Day Remembrance.
Scientists have shared details of how they are going about updating foodborne infection figures that will be published by the World Health Organization (WHO) in 2025. As part of the process to update estimates on the burden of foodborne diseases published in 2015, WHO is conducting a global source attribution... Continue Reading