Wed.Sep 11, 2024

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Gene therapy restores vision in first-of-its-kind trial

Drug Discovery World

A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. Patients who received the highest dose of the gene therapy saw up to a 10,000-fold improvement in their light sensitivity, were able to read more lines on an eye chart, and improved in their ability to navigate a standardised maze.

Therapies 173
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Microscale robot folds into 3D shapes and crawls

Science Daily: Pharmacology News

Researchers have created microscale robots less than 1 millimeter in size that are printed as a 2D hexagonal 'metasheet' but, with a jolt of electricity, morph into preprogrammed 3D shapes and crawl.

Research 134
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Industry/research collaboration to automate human brain studies 

Drug Discovery World

The Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, a neuroscience research institute based in Houston, has advanced its research capabilities with the implementation of an automation platform for human cellular model generation, maintenance, and characterisation, in collaboration with Automata Technologies.

Research 147
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Ancient DNA from Rapa Nui (Easter Island) refutes best-selling population collapse theory

Science Daily: Pharmacology News

Rapa Nui (Easter Island) with its gigantic statues and treeless landscape has fascinated researchers for centuries. A new genetic study disproves the popular theory that the Rapanui population collapsed as a result of an 'ecocide' and shows that the Rapanui admixed with Indigenous Americans centuries before Europeans arrived on the island.

DNA 124
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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Charles River hosts drug discovery seminars

Drug Discovery World

Charles River Laboratories is hosting two drug discovery seminars in the UK in September. The first will be in Cambridge on 25 September covering drug discovery capabilities, with the second at the company’s Portishead, UK site covering immuno-oncology, infectious disease capabilities on 26 September. Both events are free of charge and will feature guest speakers, networking time, refreshments, equipment demos and lab tours.

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It's High Number of Guns, Not Mental Health Crises, That Drives U.S. Gun Deaths: Study

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- Widespread and easy access to guns is the reason behind the shockingly high rate of firearm deaths in the United States, and not any rise in mental health problems suffered by perpetrators, a new study shows.The United.

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Weight-Loss Pill Saxenda Helps Kids as Young as 6

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- The weight-loss drug liraglutide helped obese children lower their BMI and reach a healthier weight, researchers report.The findings, published Tuesday in the New England Journal of Medicine and presented.

Research 116
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The latest advancements driving neuroscience drug discovery research

Drug Discovery World

In this webinar on 30 September, from 3-4PM BST, hosted by DDW and sponsored by ApconiX, you will hear from Dr Isaac Klein, Chief Scientific Officer at Dewpoint Therapeutics, Dr Sam Clark, CEO of Terran Biosciences, and Professor Ruth Roberts, Co-founder and Director of Safety Science at ApconiX. Dr Klein shares how Dewpoint applies insights into the subcellular organisation of biochemical processes into membrane-less compartments to discover novel drug targets and revolutionary drugs for diseas

Research 130
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More Women Underwent Surgical Sterilization After Fall of Roe v. Wade

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- As fears of the consequences of an unintended pregnancy rose after Roe v. Wade was overturned in 2022, rates of surgical sterilization rose in those states most affected by the decision, new research shows. Rates of.

Research 116
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Risky play exercises an ancestral need to push limits

Science Daily: Pharmacology News

Since their invention in the 1920s, jungle gyms and monkey bars have become both fixtures of playgrounds and symbols of childhood injury that anxious caretakers want removed. Anthropologists mark 100 years of the iconic playground equipment by arguing that risky play exercises a biological need passed on from apes and early humans for children to independently test and expand their physical and cognitive abilities in a context in which injury is possible but avoidable.

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.

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CAR-T Therapy Won't Raise Odds for a Second Cancer, Study Finds

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- Contrary to a warning placed on labels for CART-T cancer therapies, use of these treatments does not appear to boost the odds for a secondary cancer later, a new study shows.Researchers at Memorial Sloan Kettering.

Therapies 111
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Scientists expand the genetic alphabet to create new proteins

Science Daily: Pharmacology News

It's a dogma taught in every introductory biology class: Proteins are composed of combinations of 20 different amino acids, arranged into diverse sequences like words. But researchers trying to engineer biologic molecules with new functions have long felt limited by those 20 basic building blocks and strived to develop ways of putting new building blocks -- called non-canonical amino acids -- into their proteins.

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CDD Vault Update (September 2024)

Collaborative Drug

Chemical Safety Information: When working in a lab, you must understand the risks associated with handling chemicals. CDD Vault now displays safety information obtained from PubChem as a helpful reminder. If available, you will find hazard pictograms next to the name at the top of the molecule page.

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FDA to Investigate Safety of Toxic Metals in Tampons

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- The U.S. Food and Drug Administration announced Tuesday that it will launch an independent review into any possible effects of toxic metals found in tampons.The announcement follows the release of a study in July that.

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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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Revised Final Guidance on Nitrosamines Offers New Recommendations for Assessment and Control

FDA Law Blog: Biosimilars

By John W.M. Claud — Last week, FDA revised one of its two guidances relating to nitrosamines, Control of Nitrosamine Impurities in Human Drugs. Nitrosamines are impurities that can form during drug manufacturing and are considered potentially potent carcinogens. One specific kind of nitrosamines called N-nitrosamine drug substance-related impurities, or NDSRIs, are especially vexing to FDA and to industry because they mimic the structure of the specific active pharmaceutical ingredients (API),

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The Neanderthals may have become extinct because of their isolated lifestyle

Science Daily: Pharmacology News

Neanderthal remains recently discovered in a cave in France support well-known theory of why the Neanderthals became extinct, researchers behind a new study say.

Research 111
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Helicopter Parenting: Spotting It, and Expert Tips to Curb It

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- It’s natural for a parent to want to protect their children and keep them from harm.But sometimes this understandable desire crosses over into “helicopter parenting,” an overbearing need to be part of a child’s every dec.

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Brain-wide decision-making dynamics discovered

Science Daily: Pharmacology News

Neuroscientists have revealed how sensory input is transformed into motor action across multiple brain regions in mice. The research shows that decision-making is a global process across the brain that is coordinated by learning. The findings could aid artificial intelligence research by providing insights into how to design more distributed neural networks.

Research 104
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Good Maternity Care Increasingly Tough to Find in U.S.

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- In the two years since the March of Dimes' last report on the state of U.S. maternity care, more than 100 hospitals nationwide have shuttered their obstetric units, leaving more new moms with literally "Nowhere to Go".

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Genes with strong impact on menopause timing also link to cancer risk

Science Daily: Pharmacology News

The team first looked at variation in data from genetic sequencing of 106,973 post-menopausal female participants in the UK Biobank study. Researchers focused on rare types of genetic changes which cause a loss of the protein, and investigated their effect on the timing of menopause. The genetic changes studied are all rare in the population, however their influence on menopause is five times greater than the impact of any previously identified common genetic variant.

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Weekly Insulin Injections As Effective for Diabetes as Daily Shots, Studies Show

Drugs.com

WEDNESDAY, Sept. 11, 2024 -- Weekly insulin shots can help control both type 1 and type 2 diabetes as well as daily injections do, a pair of clinical trials have found.A new class of insulin called efsitora alfa has been designed to require.

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Keypoint Newsletter: Health Equity Recap

keypoint

By Shannon Weiman In 2023 we launched a new initiative to add health equity programming to select meetings to raise awareness for health disparities in various fields and initiate discussions on how to rectify these health inequities through scientific research and discovery. These messages are of critical importance to the global research community, so we are releasing the audio recordings of these Health Equity Forums open access, to inform broader audiences about these issues and inspire them

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Light pollution a new Alzheimer's risk factor

Science Daily: Pharmacology News

Outdoor light at night could be a significant risk factor in Alzheimer's disease, according to new research from Rush.

Disease 119
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Keypoint Newsletter: September 2024

keypoint

Checkout Health Equity highlights, climate & sustainability conferences, emerging topics & more from Keystone Symposia in our September 2024 Keypoint Newsletter.

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Mirror, mirror, in my tank, who's the biggest fish of all?

Science Daily: Pharmacology News

Researchers have demonstrated that bluestreak cleaner wrasse (Labroides dimidiatus) checked their body size in a mirror before choosing whether to attack fish that were slightly larger or smaller than themselves, saying it was the first time for a non-human animal to be demonstrated to possess some mental states that are elements of private self-awareness.

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Leveraging Nanotechnology in Generic Drug Development: Enhancing Efficacy and Safety

Drug Patent Watch

The pharmaceutical industry has witnessed significant advancements in recent years, particularly in the realm of nanotechnology. This innovative field has revolutionized the way drugs are developed, manufactured, and delivered to patients.

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Turning seawater into fresh water through solar power

Science Daily: Pharmacology News

Researchers designed an energy-efficient device that produces drinking water from seawater using an evaporation process driven largely by the sun.

Research 103
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Keypoint Newsletter: 2024 Season Highlights

keypoint

In June we wrapped up our 2024 conference season, aligning with the academic calendar. Altogether we hosted 52 conferences across North America, Europe and Asia, serving nearly 9,000 participants and providing financial aid to over 500 students, post-docs, underrepresented scientists and scientists from low- and middle-income countries. Find out more about our attendees below and see what they are saying about Keystone Symposia!

Science 85
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Atypical metabolite levels at birth may increase SIDS risk

Science Daily: Pharmacology News

Newborns who had an atypical pattern of metabolites were more than 14 times as likely to die of sudden infant death syndrome (SIDS), compared to infants who had more typical metabolic patterns, according to a new study. Metabolites are molecules produced by the body's various chemical reactions. Researchers found that infants who died of SIDS had a specific pattern of metabolites compared to infants who lived to their first year.

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BridgeBio trims gene therapy budget after seeing data on adrenal gland medicine

BioPharma Drive: Drug Pricing

Data from an early trial of the therapy fell short of the company’s “threshold to warrant additional capital investment,” an executive said.

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Astronomers track bubbles on star's surface in most detailed video yet

Science Daily: Pharmacology News

Astronomers have captured images of a star other than the Sun in enough detail to track the motion of bubbling gas on its surface. The images of the star, R Doradus, were obtained in July and August 2023. They show giant, hot bubbles of gas, 75 times the size of the Sun, appearing on the surface and sinking back into the star's interior faster than expected.

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Keypoint Newsletter: Fellows Community Updates & Call for Applications

keypoint

By Heather Gerhart Keystone Symposia’s flagship Fellows Program is now accepting applications for the Class of 2025! This unique, cutting-edge, life science Fellowship is designed to support the career development of postdoctoral and early-career scientists from underrepresented and other disadvantaged backgrounds who are engaged in biological or biomedical sciences research.

Science 67
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Clovis people used Great Lakes camp annually 13,000 years ago

Science Daily: Pharmacology News

The earliest humans to settle the Great Lakes region likely returned to a campsite in southwest Michigan for several years in a row, according to a new study.

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Dupixent succeeds in chronic hives study, giving Sanofi, Regeneron a chance to rebound

BioPharma Drive: Drug Pricing

Following an FDA rejection last year, the drugmakers plan to resubmit their application for an expanded approval of Dupixent in chronic spontaneous urticaria.

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