Thu.May 15, 2025

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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment. The process, from diagnosis to treatment, took only six months and marks the first time the technology has been successfully deployed to treat a human patient.

Treatment 281
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Alzheimer’s Drug, Leqembi, Can Be Safely Administered In Memory Clinics, Study Says

Drugs.com

THURSDAY, May 15, 2025 — The 2023 approval of the first drug shown to slow the progression of Alzheimer’s disease came with no small amount of concern from skeptics. Lecanemab (Leqembi) modestly slows Alzheimer’s by helping the.

Drugs 275
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Cyberbullying in any form can be traumatizing for kids

Science Daily: Pharmacology News

New research shows that cyberbullying should be classified as an adverse childhood experience due to its strong link to trauma. Even subtle forms -- like exclusion from group chats -- can trigger PTSD-level distress. Nearly 90% of teens experienced some form of cyberbullying, accounting for 32% of the variation in trauma symptoms. Indirect harassment was most common, with more than half reporting hurtful comments, rumors or deliberate exclusion.

Research 234
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A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases

BioPharma Drive: Drug Pricing

A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months.

Disease 319
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Bridging Innovation & Patient Care: The Growing Role of AI

Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health

AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis. But its impact goes far beyond research. As AI-driven innovation reshapes the clinical trial process, it’s also influencing broader healthcare trends, from personalized medicine to patient outcomes. Join this new webinar featuring Simran Kaur for an insightful discussion on what all of this means for the future of healthcare!

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World's largest bat organoid platform paves the way for pandemic preparedness

Science Daily: Pharmacology News

Until now, most research has used either generalized cell samples or organoids made from just one type of tropical fruit bat, and only from a single organ. But a breakthrough has arrived: a research team has now created the world's most comprehensive bat organoid platform. These 'mini-organs' are grown from five common bat species found across Asia and Europe and represent four different organs -- airway, lungs, kidneys, and small intestine.

Research 246
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FDA delays approval decision for Biohaven rare disease drug

BioPharma Drive: Drug Pricing

Shares of the biotech fell around 15% on news the FDA is not only taking longer to review the drug, but will assemble an outside group of advisers to evaluate it as well.

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AbbVie stakes $335M on a startup’s RNAi drugs

BioPharma Drive: Drug Pricing

ADARx Pharmaceuticals will work with AbbVie to make RNA interference medicines for a variety of disease areas, including neurology, immunology and cancer.

RNA 162
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Impact of oft-overlooked cell in brain function revealed

Science Daily: Pharmacology News

An often-ignored type of cell in the brain plays a dynamic and surprisingly complex role in our ability to process information, according to new research.

Research 196
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Leniolisib

New Drug Approvals

Leniolisib CAS 1354690-24-6 WeightAverage: 450.466 Monoisotopic: 450.199108558 Chemical FormulaC 21 H 25 F 3 N 6 O 2 CDZ-173-NX CDZ173 CDZ173-NX 1-[(3S)-3-({6-[6-methoxy-5-(trifluoromethyl)pyridin-3-yl]-5H,6H,7H,8H-pyrido[4,3-d]pyrimidin-4-yl}amino)pyrrolidin-1-yl]propan-1-one FDA APPROVED Joenja , 3/24/2023, To treat activated phosphoinositide 3-kinase delta syndrome Drug Trials Snapshot Leniolisib (INN [3] [4] ), sold under the brand name Joenja , is a medication used for the treatment of acti

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Key player in childhood food allergies identified: Thetis cells

Science Daily: Pharmacology News

Thetis cells, a class of immune cells first described in 2022, play an essential and previously unknown role in suppressing inflammatory responses to food, a new study finds.

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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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Celebrating Global Accessibility Awareness Day (GAAD)

Perficient: Drug Development

Today, we celebrate Global Accessibility Awareness Day (GAAD) , an annual event dedicated to raising awareness about digital accessibility and inclusion for people with disabilities. Held every third Thursday in May, GAAD encourages individuals, businesses, and organizations to reflect on the importance of creating accessible digital experiences. While GAAD highlights the needs of people with disabilities, accessibility benefits everyone not just those with disabilities.

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Digital reconstruction reveals 80 steps of prehistoric life

Science Daily: Pharmacology News

A dinosaur's 40-second journey more than 120 million years ago has been brought back to life by a research team using advanced digital modelling techniques.

Research 169
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Drug Patent Research: Expert Tips for Using the FDA Orange and Purple Books

Drug Patent Watch

"Unlocking the Secrets of the FDA's Orange and Purple Books: Insider Tips from a Drug Patent Research Expert As the pharmaceutical industry continues to evolve at breakneck speed, staying ahead of the curve requires more than just a keen eye for innovation it demands a deep understanding of the regulatory landscape. For those in the know, the FDA's Orange and Purple Books hold the key to unlocking the secrets of the industry's most closely guarded information.

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Asians made humanity's longest prehistoric migration and shaped the genetic landscape in the Americas

Science Daily: Pharmacology News

An international genomics study has revealed that early Asians undertook humanity's longest known prehistoric migration. These early humans, who roamed the earth over 100,000 years ago, are believed to have traveled more than 20,000 kilometers on foot from North Asia to the southernmost tip of South America. Scientists have mapped the unexpectedly vast genetic diversity of Asians, who make up more than half of the world's population.

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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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HP&M’s Sophia Gaulkin to Present at Life Sciences Pricing & Contracting USA Summit

FDA Law Blog: Biosimilars

Hyman, Phelps & McNamara, P.C. is pleased to announce that Sophia Gaulkin will be presenting and speaking on an expert panel at Informa Connectsannual Life Sciences Pricing & Contracting USA Summit , which is being held virtually and in-person in Philadelphia on May 19-21. Ms. Gaulkins presentation, Product Price Increases: A SPTR Practical Walkthrough , will provide an interactive case study outlining the hypothetical enforcement, appeal, and settlement scenarios that may apply to quali

Science 59
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Two HIV vaccine trials show proof of concept for pathway to broadly neutralizing antibodies

Science Daily: Pharmacology News

A decades-long scientific challenge in HIV vaccine development has been finding a way to train the immune system to produce antibodies that can target many variants of the virus. Traditional approaches haven't worked -- largely because HIV mutates rapidly and hides key parts of itself from the immune system. Now, a new study combining data from two separate phase 1 clinical trials shows that a targeted vaccine strategy can successfully activate early immune responses relevant to HIV, and, in one

Vaccine 259
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Innovative Breakthrough in Cancer Testing Developed in Ohio

Fierce BioTech

In this Fierce Biotech discussion, Chris Hayden speaks with Arnon Chait, PhD, co-founder and CEO of Cleveland Diagnostics, and Tyler Allchin, managing director of healthcare at JobsOhio. | Cleveland Diagnostics redefines cancer detection with new technology developed at the Cleveland Clinic in the heart of the Ohio Discovery Corridor.

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Ultraviolet data from NASA's Europa Clipper mission

Science Daily: Pharmacology News

The Ultraviolet Spectrograph (UVS) aboard NASA's Europa Clipper spacecraft has successfully completed its initial commissioning following the October 14, 2024, launch. Scheduled to arrive in the Jovian system in 2030, the spacecraft will orbit Jupiter and ultimately perform repeated close flybys of the icy moon Europa. Previous observations show strong evidence for a subsurface ocean of liquid water that could host conditions favorable for life.

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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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The IND Application Process: Strategic Advantages & Enhanced Opportunities

Conversations in Drug Development Trends

The Investigational New Drug (IND) application is a pivotal step in the drug development journey, offering a multitude of strategic advantages and enhanced opportunities. Have you considered the multiple associated benefits and implications in the context of your novel drug development plan? This blog highlights some of the key themes discussed in our white paper on the application process, From Concept to Market: The Strategic Role of an IND in Drug DevelopmentMoving Beyond “May Proceed.&

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Stretched in a cross pattern: Our neighboring galaxy is pulled in two axes

Science Daily: Pharmacology News

Researchers have discovered that Cepheid variable stars in our neighboring galaxy, the Small Magellanic Cloud, are moving in opposing directions along two distinct axes. They found that stars closer to Earth move towards the northeast, while more distant stars move southwest. This newly discovered movement pattern exists alongside a northwest-southeast opposing movement that the scientists previously observed in massive stars.

Research 223
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De-Risking Tech Transfer: A Smarter Path to Faster, More Reliable Outsourcing Outcomes

Fierce BioTech

| Tech transfer is conceptually a straightforward process, but it is fraught with minutiae and unknowns that can introduce delays, unplanned costs, and batch failures.

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Dual associations with two fungi improve tree fitness

Science Daily: Pharmacology News

When trees and soil fungi form close associations with each other, both partners benefit. Many tree species have further enhanced this cooperation by forming a concurrent symbiosis with two different groups of mycorrhizal fungi. Those trees cope better with water and nutrient scarcity, which is an important trait for forestry in the face of climate warming.

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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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Commissioner Makary Charts a New Course for FDA at FDLI Annual Conference

FDA Law Blog: Biosimilars

By John W.M. Claud & Deborah L. Livornese Dr. Marty Makary took the stage on Thursday at the Food and Drug Law Institutes Annual Conference , continuing the tradition of Commissioners speaking at this event, but with a tone and tempo distinctly his own. Now firmly in the chair after the agencys controversial RIFswhich he was quick to remind everyone he did not initiateCommissioner Makary said that the FDAs future depends on rebuilding its culture, empowering its staff, and applying his visi

FDA 59
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World's rivers remapped to improve flood modeling

Science Daily: Pharmacology News

A team has created the most complete map of the world's rivers ever made offering a major leap forward for flood prediction, climate risk planning, and water resource management in a warming world. The new study introduces GRIT -- a mapping system that finally shows how rivers really flow, branch, and connect landscapes.

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Broken Heart Syndrome Remains A Killer, Especially For Men

Drugs.com

THURSDAY, May 15, 2025 Broken heart syndrome sounds like a romantic, fairy-tale notion the idea that suffering a devastating loss that can cause ones heart to wither.But this syndrome, formally known as Takotsubo cardiomyopathy, continues to.

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AI overconfidence mirrors human brain condition

Science Daily: Pharmacology News

Agents, chatbots and other tools based on artificial intelligence (AI) are increasingly used in everyday life by many. So-called large language model (LLM)-based agents, such as ChatGPT and Llama, have become impressively fluent in the responses they form, but quite often provide convincing yet incorrect information. Researchers draw parallels between this issue and a human language disorder known as aphasia, where sufferers may speak fluently but make meaningless or hard-to-understand statement

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Alzheimer’s Drug Can Be Safely Administered In Memory Clinics, Study Says

Drugs.com

THURSDAY, May 15, 2025 The 2023 approval of the first drug shown to slow the progression of Alzheimers disease came with no small amount of concern from skeptics.Lecanemab (Leqembi) modestly slows Alzheimers by helping the body flush out amylo.

Drugs 243
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New gene editor enables greater precision

Science Daily: Pharmacology News

A new gene editor may soon open the door to gene therapies for a wider array of diseases.

Therapies 187
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Smartphones Aid Recovery From Broken Leg, Hip

Drugs.com

THURSDAY, May 15, 2025 A persons smartphone can show how well theyll recover from a broken leg or hip, a new study says.Smartphone data showing a persons mobility prior to their injury provides a clear window into their chances of quick recover.

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Study reveals a deep brain region that links the senses

Science Daily: Pharmacology News

Humans perceive and navigate the world around us with the help of our five senses: sight, hearing, touch, taste and smell. And while scientists have long known that these different senses activate different parts of the brain, a new study indicates that multiple senses all stimulate a critical region deep in the brain that controls consciousness. The study sheds new light on how sensory perception works in the brain and may fuel the development of therapies to treat disorders involving attention

Therapies 172
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U.S. Suspends Live Animal Imports Amid Screwworm Outbreak in Mexico

Drugs.com

THURSDAY, May 15, 2025 The U.S. Department of Agriculture (USDA) has temporarily halted the import of live cattle, horses and bison from Mexico. The move aims to stop the spread of New World screwworm, a dangerous flesh-eating parasite that can b.

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Researchers develop new metallic materials using data-driven frameworks and explainable AI

Science Daily: Pharmacology News

Found in knee replacements and bone plates, aircraft components, and catalytic converters, the exceptionally strong metals known as multiple principal element alloys (MPEA) are about to get even stronger through to artificial intelligence. Scientists have designed a new MPEA with superior mechanical properties using a data-driven framework that leverages the supercomputing power of explainable artificial intelligence (AI).

Research 168