June, 2023

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Researchers uncover new CRISPR-like system in animals that can edit the human genome

Broad Institute

Researchers uncover new CRISPR-like system in animals that can edit the human genome By Leah Eisenstadt June 28, 2023 Breadcrumb Home Researchers uncover new CRISPR-like system in animals that can edit the human genome The first RNA-guided DNA-cutting enzyme found in eukaryotes, Fanzor could one day be harnessed to edit DNA more precisely than CRISPR/Cas systems.

RNA 139
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Octopuses rewire their brains to adapt to seasonal temperature shifts

Science Daily: Pharmacology News

Octopuses don't thermoregulate, so their powerful brains are exposed to -- and potentially threatened by -- changes in temperature. Researchers report that two-spot octopuses adapt to seasonal temperature shifts by producing different neural proteins under warm versus cool conditions. The octopuses achieve this by editing their RNA, the messenger molecule between DNA and proteins.

RNA 126
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BioMarin finally secures FDA approval of hemophilia gene therapy

BioPharma Drive: Drug Pricing

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

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Getting Real with Molecular Property Prediction

Practical Cheminformatics

Introduction If you believe everything you read in the popular press, this AI business is easy. Just ask ChatGPT, and the perfect solution magically appears. Unfortunately, that's not the reality. In this post, I'll walk through a predictive modeling example and demonstrate that there are still a lot of subtleties to consider. In addition, I want to show that data is critical to building good machine learning (ML) models.

Drugs 130
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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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AHA News: Beat the Heat With Hydrating Foods This Summer

Drugs.com

TUESDAY, June 20, 2023 (American Heart Association News) -- Staying hydrated can help you live a longer, healthier life. But drinking multiple glasses of water throughout the day isn't for everyone. Health experts say that's OK. While fluids are.

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Puerto Rico on Gene & Cell Therapy: An Unparalleled Proposal for Non-Dilutive Funding

Fierce BioTech

Puerto Rico: A Hub of Opportunity for Non-Dilutive Funding | Puerto Rico is becoming the best place in the world to conduct R&D activities for emerging technologies. Could your organization maximize growth potential by capitalizing on Puerto Rico’s unique tax incentives & culture of innovation?

Therapies 119

More Trending

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Protecting Workers and the Public from Wildfire Smoke

NIOSH Science Blog: Drugs

The Canadian wildfires highlight the hazard of wildfire smoke, an issue faced annually by many communities across the United States. In this blog, the Centers for Disease Control and Prevention’s National Institute for Occupational Safety and Health (NIOSH) and National Center for Environmental Health teamed up with the Environmental Protection Agency (EPA) to provide employers, workers, and the general public information to help stay protected from wildfire smoke.

Disease 119
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First Duchenne gene therapy approved by FDA for young children

BioPharma Drive: Drug Pricing

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

Therapies 130
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Clean, sustainable fuels made 'from thin air' and plastic waste

Science Daily: Pharmacology News

Researchers have demonstrated how carbon dioxide can be captured from industrial processes -- or even directly from the air -- and transformed into clean, sustainable fuels using just the energy from the Sun.

Research 117
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When Your Child Has Scoliosis: Symptoms & Treatments

Drugs.com

FRIDAY, June 23, 2023 -- The future for a child diagnosed with scoliosis, an abnormal curvature of the spine that typically occurs just before puberty, can feel uncertain. As a parent, you want answers to your questions about scoliosis and how it.

Treatment 119
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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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Women in STEM: An Inspiring Journey with Dr Jo Brewer

Drug Target Review

Can you tell us about your journey in the field of science, technology, engineering and maths (STEM) and the challenges you encountered along the way? At school I was encouraged to follow science because I was good at it. It felt natural to follow what I was good at, and they pushed everybody to be the best they could be. That’s how I was brought up.

Therapies 115
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How chromosome imbalances can drive cancer

Broad Institute

How chromosome imbalances can drive cancer By Corie Lok June 28, 2023 Breadcrumb Home How chromosome imbalances can drive cancer An analysis of nearly 11,000 human cancers revealed how large chromosomal changes, called aneuploidies, can promote or slow the growth of tumors. By Sarah C.P. Williams June 28, 2023 Credit: Ricardo Job-Reese, Broad Communications The vast majority of cancer cells have too few or too many copies of some chromosomes, a state known as aneuploidy.

DNA 122
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Novel Oncology Target Claudin 6 Makes its Case in Solid Tumors

Fierce BioTech

Harnessing the Power Within | In the ongoing search for new cancer treatments, companies including Context Therapeutics are developing potential therapies focused on the emerging novel oncology target Claudin 6.

Therapies 111
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After years of disappointment, cancer vaccines show new promise

BioPharma Drive: Drug Pricing

Moderna presented new data at ASCO for its melanoma shot, highlighting progress with a personalized approach that’s also being pursued by BioNTech and Gritstone.

Vaccine 130
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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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Accelerating Headless Commerce with Perficient’s SAP Commerce Composable Storefront Accelerator

Perficient: Drug Development

Headless Commerce: An Innovative Strategy for E-commerce Success In the highly competitive e-commerce landscape, businesses are constantly striving to deliver exceptional digital experiences to their customers. One innovative strategy that is rapidly gaining traction is headless commerce. By decoupling the front-end presentation layer from the back-end commerce logic, headless commerce enables organizations to operate with heightened agility, facilitating faster time-to-market through agile deve

Marketing 105
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Most Women With Early Breast Cancer Will Become Long-Term Survivors, New Study Shows

Drugs.com

WEDNESDAY, June 14, 2023 -- Most women diagnosed with early breast cancer will become long-term survivors, according to new research that finds a substantial reduction in the risk of death since the 1990s. This news should reassure both patients and.

Research 119
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The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging. Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies.

Disease 111
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New image-based cellular profiling tool peers deeply into metabolic biology

Broad Institute

New image-based cellular profiling tool peers deeply into metabolic biology By Makenzie Kohler June 30, 2023 Breadcrumb Home New image-based cellular profiling tool peers deeply into metabolic biology LipocyteProfiler captures disease-relevant phenotypes in an inexpensive microscopy assay By Makenzie Kohler June 30, 2023 Credit: Phil Kubitz, Claussnitzer lab Fat cells, or adipocytes, with stains highlighting nuclei (blue); mitochondria (red); lipid droplets (green); and the actin cytoskeleton, p

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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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Do hummingbirds drink alcohol? More often than you think

Science Daily: Pharmacology News

Animals that eat fruit or sip nectar often ingest alcohol because naturally occurring yeasts turning sugar into ethanol. But how do animals feel about that? A new study details an experiment to determine whether hummingbirds are turned off by alcohol in sugar water. At 1% by volume, no. At 2% by volume, they consume much less. The implication is that hummingbirds have adjusted to small amounts of alcohol likely present in flowers and backyard feeders.

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FDA sets decision dates for Vertex, CRISPR gene editing drug

BioPharma Drive: Drug Pricing

The regulator will issue separate verdicts on the pioneering treatment’s use in sickle cell disease and beta thalassemia, with the first expected late this year.

FDA 128
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Import Data From Merlin to Finish Your Migration to Sitecore

Perficient: Drug Development

In previous posts, I shared my journey with Sitecore Data Exchange Framework and using Merlin to migrate your site to Sitecore. Learning how to use Merlin was only one part of migrating a site. In this post, I’ll share my powershell script to pull in data from Merlin and populate your content tree. I’ll continue to use the blogs.perficient.com site to show some examples.

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Experts Warn of Heart Dangers From Smoke of Canadian Wildfires

Drugs.com

THURSDAY, June 8, 2023 -- As a huge plume of smoke from over 400 Canadian wildfires swept south and turned New York City into a landscape that resembled Mars more than Earth, heart experts warned that air pollution can damage your heart as much as.

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Drug Target Review Cell and Gene Therapy eBook 2023

Drug Target Review

This ebook delves into the intricate world of cancer therapies, immune system breakthroughs, therapeutic genetic engineering, and groundbreaking advancements in gene editing, featuring insightful interviews with renowned experts in the field. The first article features an exclusive interview with Dr John Maher, Chief Scientific Officer of Leucid Bio, as he addresses the complex hurdles encountered during the development of CAR T-cell therapy for solid tumours.

Therapies 105
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Scientists pinpoint where thousands of individual proteins are made in intact tissue and single cells

Broad Institute

Scientists pinpoint where thousands of individual proteins are made in intact tissue and single cells By Corie Lok June 29, 2023 Breadcrumb Home Scientists pinpoint where thousands of individual proteins are made in intact tissue and single cells A new technology called RIBOmap can give researchers valuable insight into how protein production in animal and human tissue is altered in disease.

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Colorful fresh foods improve athletes' vision

Science Daily: Pharmacology News

Nutrition is an important part of any top athlete's training program. And now, a new study proposes that supplementing the diet of athletes with colorful fruits and vegetables could improve their visual range. The paper examines how a group of plant compounds that build up in the retina, known as macular pigments, work to improve eye health and functional vision.

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FDA panel backs full approval for Eisai, Biogen Alzheimer’s drug

BioPharma Drive: Drug Pricing

In a 6-0 vote, a group of independent advisers agreed recent clinical trial data confirmed the drug’s benefit, paving the way for an FDA decision early next month.

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What is celiac disease?

Antidote

Celiac disease is a common autoimmune disorder that causes a reaction to gluten within the small intestine. It is often underdiagnosed, but it is estimated to impact around 2 million people in the United States.

Disease 98
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Don't Get (Sun)Burned This 4th of July Weekend

Drugs.com

FRIDAY, June 30, 2023 -- Adding sun protection to your holiday gatherings takes little effort and can help you avoid the pain and potential side effects of sunburn. As Americans prepare to celebrate July 4 with parades, swimming and cookouts, the.

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Drug Target Review Imaging eBook

Drug Target Review

In this eBook, embark on an exploration of how cutting-edge imaging modalities can elevate your biomedical research. As we delve into an exclusive article with Dr Haley Marks, biomedical engineer, and project scientist at CNSI in UCLA, and her exceptional team. Additionally, gain valuable insights from Victoria Goldenshtein, PhD candidate in Biomedical Engineering at Duke University, on the ground breaking in vitro library display platform for directed protein evolution, known as GRIP Display.

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Researchers identify gut microbiome signatures linked to precancerous colon polyps

Broad Institute

Researchers identify gut microbiome signatures linked to precancerous colon polyps By Tom Ulrich June 14, 2023 Breadcrumb Home Researchers identify gut microbiome signatures linked to precancerous colon polyps New study looks at gut microbes' influence, findings may help with prevention By MGH News And Public Affairs June 14, 2023 Credit: NCI Center for Cancer Research Human colon cancer cells.

Research 105
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Gullies on Mars could have been formed by recent periods of liquid meltwater, study suggests

Science Daily: Pharmacology News

A study offers new insights into how water from melting ice could have played a recent role in the formation of ravine-like channels that cut down the sides of impact craters on Mars.

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UniQure stock tumbles on ‘confusing’ results for Huntington’s gene therapy

BioPharma Drive: Drug Pricing

While treated patients appear to be doing better than history suggests they would, mixed findings involving a key surrogate marker perplexed Wall Street analysts and sent shares falling 38%.

Therapies 120
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What motivates patients to participate in clinical trials?

Antidote

In order to connect people with medical research opportunities, it is important to understand what motivates patients to participate in clinical trials. We asked 4,000 volunteers and caregivers to gain insight into their motivation for clinical trial participation. Here’s what we learned.