Sat.May 04, 2024 - Fri.May 10, 2024

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Phase II/III study in schizophrenia patients show positive top-line results 

Drug Discovery World

Newron Pharmaceuticals has announced positive top-line results from its potentially pivotal study 008A, evaluating the safety, tolerability and efficacy of evenamide (30 mg bid) in patients with chronic schizophrenia currently being treated with a second-generation antipsychotic including clozapine, but demonstrating an inadequate response to that treatment.

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Participants of pioneering CRISPR gene editing trial see vision improve

Science Daily: Pharmacology News

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a new article.

Trials 141
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Patient dies in Pfizer study of Duchenne gene therapy

BioPharma Drive: Drug Pricing

Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly. The company is working with trial researchers to investigate further.

Therapies 144
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There's a New Set of COVID Variants Called FLiRT: What You Need to Know

Drugs.com

WEDNESDAY, May 8, 2024 -- The virus behind COVID has mutated again, this time producing variants nicknamed FLiRT, the U.S. Centers for Disease Control and Prevention (CDC) has reported.The variants are appearing in wastewater sampling, the CDC.

Virus 130
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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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Plant-based drug can help people quit vaping, study shows 

Drug Discovery World

A plant-based medication called cytisinicline may be an effective therapy to help them stop vaping, according to the ORCA-V1 trial results in the Journal of the American Medical Association (JAMA) Internal Medicine. Cytisinicline, also known as cytisine, baptitoxine, or sophorine, is an alkaloid that occurs naturally in several plant genera and has been used medically in relation to smoking cessation.

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AI systems are already skilled at deceiving and manipulating humans

Science Daily: Pharmacology News

Many artificial intelligence (AI) systems have already learned how to deceive humans, even systems that have been trained to be helpful and honest. Researchers describe the risks of deception by AI systems and call for governments to develop strong regulations to address this issue as soon as possible.

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Americans Got Drug-Resistant Infections After Stem Cell Treatments in Mexico

Drugs.com

THURSDAY, May 9, 2024 -- Antibiotic-resistant meningitis or severe, long-lasting joint infections: That's what three U.S. "medical tourists" brought home after seeking out unapproved stem cell treatments in Mexico, according to a new report.The.

Treatment 118
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Funding of $85 million secured for Prader-Willi Syndrome drug

Drug Discovery World

Aardvark Therapeutics has announced an $85 million oversubscribed Series C financing. The funding will be used to complete the clinical trials required for regulatory approval of Aardvark’s lead asset, ARD-101, for the treatment of hyperphagia in patients with Prader-Willi Syndrome (PWS), as well as to demonstrate ARD-101’s complementary mechanism of action to the current GLP-1 therapies in the treatment of obesity, and to advance other Aardvark pipeline programmes.

Drugs 173
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A fragment of human brain, mapped in exquisite detail

Science Daily: Pharmacology News

Researchers have created the largest synaptic-resolution, 3D reconstruction of a piece of human brain to date, showing in vivid detail each cell and its web of neural connections in a piece of human temporal cortex about half the size of a rice grain.

Research 132
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AstraZeneca withdraws COVID-19 vaccine, citing declining demand

BioPharma Drive: Drug Pricing

The move ends a turbulent saga for AstraZeneca, which successfully developed a coronavirus shot but struggled to sell it amid competition and the emergence of rare but serious side effects.

Vaccine 120
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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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Scientists Developing Vaccine Against Present and Future COVID Viruses

Drugs.com

MONDAY, May 6, 2024 -- Scientists are busy working on a vaccine that might fight strains of the COVID virus SARS-CoV-2 that haven't even emerged yet.The effort from a British team at the University of Cambridge is already showing promise in mouse.

Virus 116
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This week in drug discovery (6-10 May) 

Drug Discovery World

News round-up for 6-10 May by DDW Multimedia Editor Megan Thomas. A leading news story this week has been the announcement of positive Phase II results of a head and neck cancer trial, but there have been a number of other interesting clinical result announcements. These include the discovery of molecular doorways that could be used to help deliver drugs into the brain to treat neurological disorders, a plant-based medication which may be an effective therapy to help patients stop vaping, immune

Drugs 173
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Possible atmosphere surrounding rocky exoplanet

Science Daily: Pharmacology News

Researchers may have detected atmospheric gases surrounding 55 Cancri e, a hot rocky exoplanet 41 light-years from Earth. This is the best evidence to date for the existence of any rocky planet atmosphere outside our solar system.

Research 127
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#ScienceSaturday: May 4, 2024

KIF1A

#ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research Clinical and Genetic Characterization of a Cohort of Brazilian Patients With Congenital Ataxia KAND is a rare disorder, but each new patient makes it slightly less so.

Science 116
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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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Gene Therapy Improves Vision in People With Inherited Blindness

Drugs.com

MONDAY, May 6, 2024 -- An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.Researchers recruited 14 people with Leber Congenital Amaurosis.

Therapies 116
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New DDW Highlights episode: 8 May 2024

Drug Discovery World

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates. The ongoing innovation and development in the drug discovery sector relies on a number of moving parts, one of which is financial support and backing.

Disease 173
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Genetics, not lack of oxygen, causes cerebral palsy in quarter of cases

Science Daily: Pharmacology News

The world's largest study of cerebral palsy (CP) genetics has discovered genetic defects are most likely responsible for more than a quarter of cases in Chinese children, rather than a lack of oxygen at birth as previously thought.

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iTeos shares jump on TIGIT update; Acelyrin swaps CEOs

BioPharma Drive: Drug Pricing

Interim data surpassed expectations, iTeos said. Elsewhere, Acelyrin revealed chief executive officer Shao-Lee Lin is departing and Bluebird bio gave a fuller account of its gene therapy launches.

Therapies 107
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Can Zinc Really Shorten a Cold?

Drugs.com

THURSDAY, May 9, 2024 -- Every cold and flu season, folks are flooded with ads for zinc lozenges, sprays and syrups that promise to shorten their sniffles.Zinc might indeed reduce the duration of common cold symptoms by about two days, a new.

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How can long-read nanopore sequencing support gene therapy delivery?

Drug Discovery World

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. The event will be supported by Oxford Nanopore Technologies. The event will feature experts Daniel Jachimowicz, Senior Bioscience Scientist at AstraZeneca BioPharmaceuticals R&D, Sweden, and Bastian Schiffthaler, Senior Data Scientist at AstraZeneca BioPharmaceuticals R&D, Sweden.

Therapies 173
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Swarms of miniature robots clean up microplastics and microbes, simultaneously

Science Daily: Pharmacology News

When old food packaging, discarded children's toys and other mismanaged plastic waste break down into microplastics, they become even harder to clean up from oceans and waterways. These tiny bits of plastic also attract bacteria, including those that cause disease. Researchers describe swarms of microscale robots (microrobots) that captured bits of plastic and bacteria from water.

Packaging 124
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Handling Not Allowed Reflection Method in Sitecore

Perficient: Drug Development

Recently, to meet project requirements, we customized and expanded the functionality of the “General Link” feature by incorporating a new “Telephone Link” feature. Everything was working correctly on our local project instance, but we got the below-listed error when the changes were deployed on the higher environment. Exception: Sitecore.Exceptions.AccessDeniedException Message: Calling Fieldtypes.ExtendedGeneralLink.ExtendedGeneralLinkForm.OnModeChange method through ref

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Combo Therapy May Be Advance Against Liver Cancer

Drugs.com

MONDAY, May 6, 2024 -- A new combination therapy appears to boost the response rate for liver cancer patients receiving immunotherapy, according to results from a phase 2 clinical trial.The combo therapy involves bavituximab, a drug that.

Therapies 112
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Positive results of Phase II head and neck cancer trial 

Drug Discovery World

Calliditas Therapeutics has announced data from the proof-of-concept Phase II trial evaluating setanaxib, its lead NOX enzyme inhibitor, in combination with pembrolizumab, in patients with squamous cell carcinoma of the head and neck (SCCHN). The analysis showed statistically significant improvements in progression-free survival (PFS), as well as in overall survival (OS), with statistically significant changes in tumour biology consistent with the mechanism of action of setanaxib.

Trials 173
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Pressure to be 'perfect' causing burnout for parents, mental health concerns for their children

Science Daily: Pharmacology News

Is the status of 'perfect parent' attainable? Researchers leading a national dialogue about parental burnout say 'no,' and a new study finds that pressure to try to be 'perfect' leads to unhealthy impacts on both parents and their children.

Research 119
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How CDISC SDTM Improves Cost & Quality in ISS/ISE in 2024

Quanticate

When investigating the efficacy and safety of a new drug/device it is sometimes necessary to combine the research from multiple clinical trials for your analysis. In such cases, it is important to consider the cost and quality of producing integrated summaries and how CDISC SDTM (Clinical Data Interchange Standards Consortium Study Data Tabulation Model) can aid in reducing costs and improving quality for Integrated Summary of Safety (ISS) and Integrated Summary of Efficacy (ISE) submissions.

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Almost All Counterfeit Oxycontin Pills Contain Fentanyl

Drugs.com

TUESDAY, May 7, 2024 -- Lab tests of counterfeit oxycodone (Oxycontin) pills seized by police in Rhode Island in 2022 found 99.3% also contained dangerous fentanyl. It was typically mixed with another potentially deadly drug, xylazine.Both.

Drugs 111
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New cell engineering partnership to accelerate biopharma innovation 

Drug Discovery World

Mekonos, a company working towards cell engineering on a chip, has entered into a partnership agreement with Accelerated Bio to codevelop a new manufacturing cell line based on Accelerated Bio’s human Trophoblast Stem Cells (hTSCs) and Gene Engineered Multi-Sites (GEMS) technology. Dr Anil Narasimha, Co-founder and Chief Executive Officer of Mekonos, said: “Our platform brings together semiconductor, microfluidic, and surface chemistry technologies to enable the full potential of gene editing

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Generative AI that imitates human motion

Science Daily: Pharmacology News

Walking and running is notoriously difficult to recreate in robots. Now, a group of researchers has overcome some of these challenges by creating an innovative method that employs central pattern generators -- neural circuits located in the spinal cord that generate rhythmic patterns of muscle activity -- with deep reinforcement learning. The method not only imitates walking and running motions but also generates movements for frequencies where motion data is absent, enables smooth transition mo

Research 117
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Can a Device Be Found Not Substantially Equivalent Because of Cybersecurity Risks? A Review of FDA’s Draft Guidance on Cybersecurity in Medical Devices

FDA Law Blog: Biosimilars

By Lisa M. Baumhardt, Senior Medical Device Regulation Expert & Adrienne R. Lenz, Principal Medical Device Regulation Expert — FDA recently issued a draft guidance which would update the agency’s Cybersecurity in Medical Devices: Quality System Considerations and Content of Premarket Submissions guidance. The draft guidance provides recommendations on what is required to meet cybersecurity obligations under section 524B of the Food, Drug and Cosmetic Act (FD&C).

FDA 105
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Baby Girl Born Deaf Gains Hearing After Gene Therapy

Drugs.com

FRIDAY, May 10, 2024 -- Opal Sandy was born into a world she could not hear. The British baby girl, now 18 months old, had a rare genetic condition called auditory neuropathy that interrupted nerve impulses that travel from the inner ear to.

Therapies 105
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AI and machine learning: A new era for clinical trials

Drug Discovery World

Fiona Maini , Senior Director Global Compliance & Strategy, at Medidata looks at the rise of artificial intelligence (AI) and machine learning (ML) within life sciences and the applications for the technology in clinical trials. The benefits that AI and machine learning (ML) can bring to society and their potential to radically change the world we live in can sometimes feel a little intangible, futuristic or even far-fetched.

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How the brain is flexible enough for a complex world (without being thrown into chaos)

Science Daily: Pharmacology News

Many neurons exhibit 'mixed selectivity,' meaning they can integrate multiple inputs and participate in multiple computations. Mechanisms such as oscillations and neuromodulators recruit their participation and tune them to focus on the relevant information.

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Antidote to be announced as Corporate Honoree at MSAA Improving Lives Benefit

Antidote

We are thrilled to be honored as the Multiple Sclerosis Association of America’s Corporate Honoree at their 10th annual Improving Lives Benefit on May 15! This inspiring event supports MSAA’s free programs and services and honors key players in the MS research space.

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