Fri.Mar 17, 2023

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FDA reversal sets up high-stakes hearing on Sarepta gene therapy

Bio Pharma Dive

The regulator will convene a panel of advisers to consult on an approval decision for the biotech’s Duchenne treatment, a change from just a few weeks ago when Sarepta had said a meeting wouldn’t be held.

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Pharmanovia signs licence deal for Aeterna Zentaris’ Ghryvelin

Pharmaceutical Technology

Pharmanovia and Aeterna Zentaris have entered an exclusive licensing agreement for the latter’s medicine Ghryvelin (macimorelin). Under the deal, Pharmanovia will acquire the exclusive rights and license to commercialise Ghryvelin in the EEA and the UK from Consilient Health. Ghryvelin is used for the diagnosis of Adult Growth Hormone Deficiency (AGHD), a rare and serious condition, as well as for Childhood Onset Growth Hormone Deficiency (CGHD), if approved.

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Pear, tight on cash, considers strategic alternatives

Bio Pharma Dive

The digital therapeutics firm is considering a sale, merger or other deal following struggles to market its app-based treatments.

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LCRF, Daiichi Sankyo and AstraZeneca enter research collaboration

Pharmaceutical Technology

The Lung Cancer Research Foundation (LCRF) has announced a new research partnership with Daiichi Sankyo and AstraZeneca. This collaboration is intended to fund up to three research grants that are focused on antibody-drug conjugates (ADCs) for improving the outcomes for lung cancer patients. ADCs are designed for targeting cancer cells specifically and selectively provide a highly potent payload that may limit healthy cells’ damage.

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The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

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JAMA’s new editor settles in, bringing open access and other changes

STAT News

ST. LOUIS — It’s been eight months since Kirsten Bibbins-Domingo, a general internist, cardiovascular researcher, and epidemiologist became the 17th editor in chief of the Journal of the American Medical Association and its network of journals. Bibbins-Domingo, who previously worked at the University of California, San Francisco, was named this week as a member of the 2023 STATUS List of people making a difference in health care and life sciences.

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US FDA approves Novartis’ Tafinlar and Mekinist combination therapy

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval for Novartis ’ combination therapy, Tafinlar (dabrafenib) and Mekinist (trametinib), to treat BRAF V600E low-grade glioma (LGG) in paediatric patients as young as one year. The liquid formulations of Tafinlar and Mekinist also received approval for ease of administration across several approved indications.

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‘I’m terrified’: Patients scramble for treatment after 13 ketamine clinics shut down

STAT News

Around 4:30 p.m. last Friday, Ketamine Wellness Centers CEO Kevin Nicholson sent an email: In 30 minutes, the company would no longer be in business, he said. The message went out to some patients and to all of the employees of his company’s clinics across nine states — what had, up until moments before, been one of the largest ketamine clinic chains in the country.

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'Back to one meal a day': SNAP benefits drop as food prices climb

NPR Health - Shots

A lot of people don't think twice about buying milk, says Teresa Calderez. "But there are lots of us out here who can't buy a gallon of milk when we need it.

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STAT+: Moderna CEO made $398 million in 2022, but still pledges to give most to charity

STAT News

Moderna CEO Stéphane Bancel is starting to reap gargantuan gains from the stock he first got when he started with the company a decade ago, although nearly all of this chunk of his fortune remains earmarked for unknown charities. Bancel made $398 million in 2022 based on the actual realized gains of stock that was exercised and sold, according to STAT’s calculations from Moderna’s annual compensation disclosure filed this week.

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Read the transcript: What happened inside the federal hearing on abortion pills

NPR Health - Shots

Read the transcript from Wednesday's hearing before Judge Matthew Kacsmaryk in a federal case that could decide the future of access to a major abortion pill.

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Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.

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Opinion: The staggering financial burden of a proposed HIPAA rule

STAT News

The already beleaguered U.S. health care system is facing a new and costly threat that will affect patient care and ultimately may lead to hospital closures : paying for and processing a torrent of medical record requests. While the news media in 2022 focused on hospitals’ billions of dollars of losses , negative operating margins, and other daunting post-pandemic challenges , a set of costly modifications to the HIPAA Privacy Rule proposed by the Department of Health and Human Services m

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Clinics on wheels bring doctors and dentists to health care deserts

NPR Health - Shots

Health care vans that provided COVID testing and vaccines in the pandemic are now providing a range of health services in hard-to-reach communities. New access to federal funds could expand the trend.

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Study examines the link between mental health and oral health

Medical Xpress

A study examining the mental health-oral health association cross-sectionally and longitudinally was presented at the 52nd Annual Meeting & Exhibition of the AADOCR, held in conjunction with the 47th Annual Meeting of the CADR. The AADOCR/CADR Annual Meeting & Exhibition took place at the Oregon Convention Center in Portland on March 15-18, 2023.

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Cyclone Freddy shattered records. People lost everything. How does the healing begin?

NPR Health - Shots

The countries hit by this unprecedented storm now must help survivors who may have lost nearly everything. We talk to an aid worker on the ground in Mozambique for insights.

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The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

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Dangerous hitchhikers: Visualizing how we spread COVID-19 within our homes

Medical Xpress

While COVID-19 can be transmitted via contact with contaminated objects, most studies have focused on airborne droplet transmission. Against this backdrop, scientists from Japan conducted detailed computational simulations based on real-life behavioral data to visualize how viruses spread to common household items shortly after people return home. Their results illustrate the importance of immediate hand sanitization upon returning to avoid contact infection, reinforcing the effectiveness of a g

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What is Babesiosis? A rare tick-borne disease is on the rise in the Northeast

NPR Health - Shots

Lyme Disease isn't the only tick-driven illness that residents of the northeast should worry about. The CDC says cases of Babesiosis, which can cause flu-like symptoms, are on the rise in 8 states.

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Study identifies key role for nervous system in severe allergic shock

Medical Xpress

A key feature of the severe allergic reaction known as anaphylaxis is an abrupt drop in blood pressure and body temperature, causing people to faint and, if untreated, potentially die.

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STAT+: To prevent emergency C-sections, researchers turn to AI to predict the course of childbirth

STAT News

After more than a decade as an OB-GYN, Darine El-Chaâr still finds late-stage emergency C-sections to be hard. Labor can drag on for days without a baby being born, at which point the situation grows dangerous. “Many times I walk to the operating room, and I’m like, I’m so sorry that you got so far and now this is where you are,” said El-Chaâr, a physician at the Ottawa Hospital Research Institute.

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Accelerating Clinical Supply Through Integrated Drug Development

As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinical trials. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.

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Study examines how our native language shapes our brain wiring

Medical Xpress

Scientists at the Max Planck Institute for Human Cognitive and Brain Sciences in Leipzig have found evidence that the language we speak shapes the connectivity in our brains that may underlie the way we think. With the help of magnetic resonance tomography, they looked deep into the brains of native German and Arabic speakers and discovered differences in the wiring of the language regions in the brain.

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STAT+: Pharmalittle: Sanofi cuts price on a popular insulin by 78%; Califf criticizes insurers for doing too little on drug research

STAT News

And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is exceedingly modest. We plan to promenade with the official mascot, catch up on our napping, and hope to have a listening party with Mrs. Pharmalot. So far, the rotation includes this , this and this.

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New gene-editing technique reverses vision loss in mice

Medical Xpress

Researchers in China have successfully restored the vision of mice with retinitis pigmentosa, one of the major causes of blindness in humans. The study, to be published March 17 in the Journal of Experimental Medicine, uses a new, highly versatile form of CRISPR-based genome editing with the potential to correct a wide variety of disease-causing genetic mutations.

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STAT+: Up and down the ladder: The latest comings and goings

STAT News

Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others.   That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going. And here is our regular feature in which we highlight a different person each week.

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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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Study examines whether replacements for missing teeth lower the risk of poor cognition

Medical Xpress

A study aiming to determine if the replacement of missing teeth with fixed prostheses may protect against cognitive decline was presented at the 52nd Annual Meeting & Exhibition of the AADOCR, in conjunction with the 47th Annual Meeting of the CADR. The AADOCR/CADR Annual Meeting & Exhibition took place at the Oregon Convention Center in Portland on March 15-18, 2023.

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With gun control far from sight, schools redesign for student safety

NPR Health - Shots

In the wake of ongoing school shootings, architects are designing schools to minimize risk and increase student connection.

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Trajectories of opioid prescribing by dentists and OMFS analyzed to identify high prescribers

Medical Xpress

A study aiming to identify high prescribers by defining trajectories of opioid prescribing among dentists and oral and maxillofacial surgeons (OMFS) was presented at the 52nd Annual Meeting & Exhibition of the AADOCR, held in conjunction with the 47th Annual Meeting of the CADR. The AADOCR/CADR Annual Meeting & Exhibition took place at the Oregon Convention Center in Portland on March 15-18, 2023.

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iOnctura reveals roginolisib research results

Pharma Times

Therapy prevents tumour proliferation while breaking immune tolerance among cancer patients

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The New Clinical Trial Supply Chain: Resilient, Flexible, and Patient-Centric

The global landscape of clinical trials is rapidly changing as studies become more complex. An increasing number of sponsors are seeking enhanced flexibility in their supply chains to address a variety of clinical supply challenges, including patient demand and reducing delays. Demand-led supply and direct-to-patient distribution are next-generation solutions that are helping to meet these growing needs, allowing for more streamlined processes and patient-centric studies.

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Study hints at the promise of non-hallucinogenic LSD for treating mood disorders

Medical Xpress

Mood disorders are mental health conditions characterized by persistently dysregulated moods, such as recurring feelings of depression or euphoria. According to statistics by the National Institutes of Health, approximately 1 in 5 people in the United States will experience mood disorders at some point in their life.

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Astellas, Pfizer look to broaden Xtandi’s patient base with new prostate cancer win after 8 years

Fierce Pharma

Astellas, Pfizer look to broaden Xtandi’s patient base with new prostate cancer win after 8 years aliu Fri, 03/17/2023 - 10:22

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Few Medicaid-participating primary care physicians providing longer-acting birth control methods, finds study

Medical Xpress

Medicaid beneficiaries face barriers in accessing medical care—and that includes contraceptive care. A new study finds that despite birth control being an essential health service, all primary care physicians that see them may not be offering Medicaid patients some of the most effective, longer-acting birth control methods. While nearly half (48%) of primary care physicians who treat Medicaid patients provided prescription contraception like the birth control pill, only 10% provided longer-actin

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Great Ormond Street Hospital and Leucid Bio collaborate

Pharma Times

GOSH will collaborate with Leucid at the Zayed Centre for Research into Rare Disease in Children

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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.