Wed.Mar 13, 2024

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STAT+: Hemophilia gene therapies arrived after 40 years of struggle. Where are the patients?

STAT

As a boy growing up with hemophilia A, Noah Frederick reserved the end of his annual checkups to talk about new technologies. His doctor walked through various experimental approaches for the bleeding disorder and, invariably, gene therapy. It was coming, he always said, in your lifetime. Last year, it finally arrived. But Frederick, now a 23-year-old software engineer, isn’t sure he’ll get it.

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These biotech hotspots are ripe for IPOs

PharmaVoice

Biotech IPOs are off to the races in 2024, and investors are betting on horses that are derisked in targeted areas.

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STAT+: Small studies offer hope CAR-T can fight an aggressive brain cancer

STAT

A series of new studies are raising hopes that CAR-T , a process in which treatments are made by genetically editing a patient’s own white blood cells, can eventually be used to treat an incurable and deadly type of brain cancer, called glioblastoma multiforme, or GBM. In the most dramatic result, from a three-person study conducted by researchers at Massachusetts General Hospital, a 72-year-old man saw his tumor shrink 18.5% in just two days and then decrease further over the next two mo

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Former deputy CPhO to investigate medicines shortages

The Pharmacist

A new group to investigate the causes of medicines shortages will be chaired by former deputy chief pharmaceutical officer (CPhO) for NHS England and Royal Pharmaceutical Society (RPS) fellow Dr Bruce Warner. The group will be convened by the RPS and will bring together experts from primary and secondary care, patients, the pharmaceutical industry, suppliers, […] The post Former deputy CPhO to investigate medicines shortages appeared first on The Pharmacist.

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Position Your Pharmacy for Expansion

Speaker: Chris Antypas and Josh Halladay

Access to limited distribution drugs and payer contracts are key to pharmacy expansion. But how do you prepare your operations to take the next step? Meaningful data: Collect and share clinical data regarding outcomes, utilization, and more Reporting: Limited distribution models require efficient tracking and reporting systems Workflows: Align workflows with specific pharma and payer contractual requirements For in-depth, expert insights on pharmacy expansion, watch this webinar from Inovalon.

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STAT+: In large trial, Guardant Health’s blood test detects colon cancer, but less reliably at earliest or precancerous stages

STAT

Doctors have been eagerly waiting for data on Guardant Health’s new blood-based colorectal cancer screening test, the Shield test. The hope has been that Guardant would be able to achieve an elusive goal in colorectal cancer screening: catch incipient or early colorectal cancer using nothing more than two vials of blood. Those hopes were partially fulfilled on Wednesday.

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Jay Badenhorst to take up new PDA director of pharmacy role

The Pharmacist

The Pharmacists' Defence Association has announced the appointment of Jay Badenhorst as its director of pharmacy – a first-of-its-kind role. The move comes as the National Pharmacy Association (NPA) today confirmed Mr Badenhorst had stepped down as its vice chair and board member at a board meeting on Tuesday. He has been replaced at the […] The post Jay Badenhorst to take up new PDA director of pharmacy role appeared first on The Pharmacist.

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More Trending

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Supporting patients with diabetes during Ramadan fasting

The Pharmacist

GP and diabetes specialist Dr Waqas Tahir explains how primary care teams can support people living with diabetes to manage their condition safely when fasting. Ramadan is the ninth month in the Islamic lunar calendar, when healthy Muslims will fast during the day (not eating or drinking from dawn until dusk). Most people take two meals […] The post Supporting patients with diabetes during Ramadan fasting appeared first on The Pharmacist.

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STAT+: AI health care companies say they’ll keep humans in the loop. But what does that actually mean?

STAT

Developers of artificial intelligence models slowly making their way into medicine have long parried ethical concerns with assertions that clinical staff must review tech’s suggestions before they are acted on. That “human in the loop” is meant to be a backstop preventing potential medical errors conjured up by a flawed algorithm from harming patients.

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Transitioning PROs from paper to digital: Maintaining data integrity and patient experience

pharmaphorum

Transitioning patient-reported outcomes (PROs) from paper to digital (ePRO) is crucial for maintaining data integrity and improving the patient experience. Learn how to seamlessly make this transition and optimise your data collection process.

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STAT+: Pharma loses a key battle over a state law concerning 340B pharmacies

STAT

In a setback to the pharmaceutical industry, a U.S. appeals court has upheld an Arkansas law that allows hospitals participating in a controversial federal discount program to ship medicines to certain pharmacies. As a result, other states can now be expected to pursue similar laws. The closely watched decision comes amid a long-running battle over the program, which was designed three decades ago to help hospitals and clinics care for low-income and rural patients.

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5 Reasons to Upgrade Your Pharmacy Management Software

Are you still using workarounds to manage your daily operations? To achieve peak performance, it's time to explore other options for specialty and infusion pharmacy software. Streamline pharmacy operations and improve clinical performance with automated processing, real-time data exchange, and electronic decision support. Download this helpful infographic to: Drive efficiency and patient adherence from referral receipt to delivery and ongoing care – all with our Pharmacy Cloud.

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Lilly adds Amazon Pharmacy to LillyDirect DTC channel

pharmaphorum

Amazon Pharmacy will provide dispensing for Eli Lilly’s recently launched direct-to-consumer LillyDirect channel, covering obesity, diabetes, and migraine drugs.

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STAT+: BIO severs ties with WuXi in the face of U.S. government concerns over China

STAT

WASHINGTON — The Biotechnology Innovation Organization, an industry trade group, is cutting ties with Chinese company WuXi in response to increasing U.S. government scrutiny of it and other Chinese companies, according to a BIO press release shared with STAT on Wednesday. It’s an about-face for a lobbying organization that recently was willing to defend WuXi against attacks, and it’s a sign that the U.S. biotechnology industry will have to make do without a company that it h

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Common Residency Interview Questions from the 2024 Match 

Board Vitals - Pharmacist

As candidates prepare for the rigorous residency interview season, it’s essential to anticipate the types of questions that may arise during these pivotal conversations. While the questions asked will vary by program and specialty, certain themes and questions tend to recur across the board, and there are some questions that appear more than others.

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Opinion: Good health care price transparency rules are at risk if Congress doesn’t act

STAT

Consider shopping for cereal in the grocery store. Buyers easily know how many grams of sugar and calories are in a serving, as well as how much the box costs, before they get to the checkout line. Yet in the U.S. health care market, this often isn’t the case. Prices are both high and highly variable as well as difficult to observe. I’m an economics professor.

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European Commission approves Pfizer's PREVENAR 20® to help protect infants and children against pneumococcal disease

World Pharma News

Pfizer Inc. (NYSE: PFE) today announced that the European Commission (EC) has granted marketing authorization for the company's 20-valent pneumococcal conjugate vaccine, marketed in the European Union under the brand name PREVENAR 20®, for active immunization for the prevention of invasive disease, pneumonia and acute otitis media caused by Streptococcus pneumoniae in infants, children and adolescents from 6 weeks to less than 18 years of age.

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STAT+: Pharmalittle: We’re reading about Amazon delivering Zepbound, hemophilia treatments, and more

STAT

Good morning, everyone, and welcome to the middle of the week. Congratulations on making it this far, and remember there are only a few more days until the weekend arrives. So keep plugging away. After all, what are the alternatives? While you ponder the possibilities, we invite you to join us for a delightful cup of stimulation. Our choice today is, once again, crème brûlée.

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Controversial price concession changes to be introduced from April

The Pharmacist

Category A reimbursement prices are set to be updated quarterly, despite protests from the sector's negotiator and concerns raised in a 2019 consultation on price concession reform. And while community pharmacies will soon receive ‘top-up payments’ when the original price concession has been deemed ‘inappropriately low’, Community Pharmacy England (CPE) said this would have a […] The post Controversial price concession changes to be introduced from April appeared first on The Pharmacist.

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Geron’s resurgence might have been short-lived

STAT

Want to stay on top of the science and politics driving biotech today?  Sign up  to get our biotech newsletter in your inbox. Good morning, everyone. Damian here with a look at why some wished-for gene therapies failed to make a splash when they came true and the words of ALS patients in the aftermath of another crushing setback.

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Sanofi India, Emcure Pharma announce exclusive distribution partnership

Express Pharma

Sanofi India and Emcure Pharmaceuticals signed an exclusive distribution and promotion agreement, effective immediately. Emcure Pharma will exclusively distribute and promote the Sanofi India’s cardiovascular (CV) range of products that include brands like Cardace, Clexane, Targocid, Lasix and Lasilactone. Sanofi India will continue to own, import, and manufacture these brands across Sanofi’s plants in India and internationally, Emcure will engage healthcare professionals and expand reach of the

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BaseCamp unveils improved protein structure prediction model

pharmaphorum

Basecamp Research launches BaseFold, a deep learning model that it says surpasses benchmark tool AlphaFold2 and should accelerate AI-based drug discovery

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IIT Kanpur study unveil insights into working mechanism of cholesterol lowering drugs

Express Pharma

A study by researchers at the Indian Institute of Technology Kanpur’s Department of Biological Sciences and Bioengineering has led to new insights into understanding how cholesterol-lowering drugs like Niacin work at a molecular level. Utilising cryogenic-electron microscopy (cryo-EM) technology, the team, led by Prof Arun K Shukla, were able to visualise the key target receptor molecule activated by Niacin and other related drugs.

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Prilenia eyes 2025 launch for Huntington’s drug pridopidine

pharmaphorum

Prilenia Therapeutics is planning to file its Huntington’s disease drug pridopidine in the EU shortly, after mixed results in a phase 3 trial.

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AbbVie’s Produodopa approved by SMC to treat Parkinson’s disease patients in Scotland

Pharma Times

The progressive neurological disorder affects approximately 12,400 people in Scotland

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Rapid pseudomonas aeruginosa detection method developed

European Pharmaceutical Review

Findings from an experimental study report the development of a rapid method for the detection of pseudomonas aeruginosa bacteria in medications. The corresponding research paper stated the key benefits of the method include its high selectivity and specificity, cost-effectiveness as well as simplicity for application. For example, the author shared that the procedure can be used in microbiological laboratories of various levels.

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Zydus launches IBYRA, the generic version of Olaparib

Express Pharma

Zydus has launched Olaparib, a PARP inhibitor, under the brand name IBYRA in India. The drug will target specific genetic mutations prevalent in certain types of cancers. Out of 14 lakh newly diagnosed cancer patients in India, nearly 2.75 lakh patients are diagnosed with breast, ovarian, prostrate and pancreatic cancers as per Globocan 2022 data. Amongst these patients, those who are diagnosed as HRD positive or with BRACA mutation need to be administered this PARP inhibitor which can delay the

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FDA warning letters highlight data integrity issues

European Pharmaceutical Review

FDA inspections Identification of data integrity deviations Of the 70 Warning Letters issued by the US Food and Drug Administration (FDA) so far in 2024, three have identified data integrity issues at pharmaceutical manufacturing sites outside the US. Specifically, these recent warning letters describe microbiology and environmental monitoring related data integrity issues.

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PulmCrit wee: Why I like central lines for GI bleed resuscitation

EMCrit Project

People on twitter absolutely hate the concept of using a central line to resuscitate a GI bleeder. This comes up a couple times per year. I think the source of this hatred is largely two-fold: A central line alone is garbage (without a Level-1 or Belmont infuser). I'll admit that. So if you're working in […] EMCrit Project by Josh Farkas.

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US study reveals age-related skin changes could contribute to melanoma metastases

Pharma Times

Around 200,000 people in the US are expected to be diagnosed with the skin cancer this year

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eClinical innovations streamline clinical trial processes: Insights from Dawn Kaminski

Outsourcing Pharma

In the fast-evolving landscape of clinical trials, technological advancements play a vital role in streamlining processes, enhancing efficiency, and ultimately improving patient outcomes.

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Bright Green invests $250m to expand facility in New Mexico, US

Pharmaceutical Technology

Bright Green has announced plans to expand its controlled substances drug production facility in New Mexico, US with an investment of $250m.

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From a childhood fascination to a pull towards translational medicine, who is this week's Woman in Science?

Outsourcing Pharma

Kate Broderick takes us on her intriguing journey - from being drawn to how science was applied within a medical setting, to the enormous challenge of working around the clock when the pandemic began.

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FDA to focus on ‘early deaths’ in meeting on broader CAR-T use in myeloma

BioPharma Dive

Briefing documents posted ahead of Friday’s advisory panel zeroed in on increased rates of early death in tests of cell therapies from Bristol Myers Squibb and Johnson & Johnson.

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Basecamp Research unveils improved protein structure model

pharmaphorum

Basecamp Research launches BaseFold, a deep learning model that it says surpasses benchmark tool AlphaFold2 and should accelerate AI-based drug discovery.

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J&J reveal long-term data for novel oral peptide

European Pharmaceutical Review

The first and only investigational targeted oral peptide has been shown to maintain high rates of skin clearance in adults with moderate-to-severe plaque psoriasis through 52 weeks, new data shows. In the long-term extension FRONTIER 2 study , JNJ-2113 demonstrated sustained efficacy from Week 16 to Week 52, based on data presented at the American Academy of Dermatology (AAD) 2024 Annual Meeting.

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FDA reviewer pokes holes in Geron’s MDS drug data

pharmaphorum

FDA reviewers seem unconvinced by Geron's data on myelodysplastic syndrome therapy imetelstat ahead of FDA adcomm meeting.

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