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Child walks again after receiving experimental treatment for rare genetic condition

STAT

In what experts are calling a “dream come true,” scientists used a recent biochemical discovery to help an 8-year-old boy with a rare genetic condition regain mobility. Researchers from NYU Langone demonstrated, in a study published in Nature on Wednesday , how a chemical precursor to a commonly available enzyme, CoQ10, can help brain cells overcome a rare genetic condition that severely hobbles cells’ energy production process.

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Pricing watchdog will take on the rising cost of drugs at launch

PharmaVoice

ICER’s upcoming report could shed light on how the launch price of newly approved drugs affects overall cost and access from a patient perspective.

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BiomX phage therapy shows potent effect in cystic fibrosis trial

Pharma Times

Subscribe Advertise About us Follow Follow Follow Follow GO Magazine PharmaTimes Magazine Archive Web Exclusives News Competitions Appointments Business Insights Webinars Thought Leadership BiomX phage therapy shows potent effect in cystic fibrosis trial by John Pinching | 9th Jul 2025 | News BX004 achieves 500-fold bacterial reduction with no resistance in phase 1b/2a study BiomX has published promising phase 1b/2a trial results in Nature Communications , demonstrating that its phage cocktail B

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Veeda Lifesciences invests in Mango Sciences to integrate AI into global clinical trial operations

Express Pharma

Veeda Lifesciences, a global contract research organisation (CRO), has announced an investment in Mango Sciences, a Boston-based healthcare AI and data company. The investment is part of Veeda’s strategy to integrate artificial intelligence into its clinical trial operations, with a focus on improving patient recruitment, trial efficiency, and diversity across global markets.

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Navigating Payroll Compliance: Future-Proofing Payroll in an Evolving Regulatory Landscape

Speaker: Jennifer Hill

Payroll compliance is a cornerstone of business success, yet for small and midsize businesses, it’s becoming increasingly challenging to navigate the ever-evolving landscape of federal, state, and local regulations. Mistakes can lead to costly penalties and operational disruptions, making it essential to adopt advanced solutions that ensure accuracy and efficiency.

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Pharmacy groups call on insurers to preserve vaccine coverage

Beckers Hospital Review

A coalition of pharmacy organizations, including the American Society of Health-System Pharmacists, has called for major U.S. health insurers to preserve existing vaccine coverage without increasing patient cost-sharing. In letters sent to major insurers including UnitedHealthcare, Humana, Cigna, Kaiser Permanente and trade groups such as AHIP and Blue Cross Blue Shield Association, the organizations expressed concerns about the potential coverage changes following updates by the CDC’s Adv

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Leading medical groups sue Kennedy over changed Covid-19 vaccine recommendations

STAT

WASHINGTON — Six major medical groups, and a pregnant physician, are suing health secretary Robert F. Kennedy Jr. over changes he made to Covid-19 vaccine recommendations that they say were unlawful and undermine public trust in health care. The suit argues that a May 19 directive signed by Kennedy, which said that the Covid-19 vaccine would no longer be recommended for healthy children and pregnant people , violates decades of policy governing how vaccines are reviewed, approved, an

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FDA approves first-of-a-kind oral therapy for rare disease

European Pharmaceutical Review

The US Food and Drug Administration (FDA) has approved the first oral on-demand therapy for acute attacks of hereditary angioedema (HAE) in individuals aged 12 years and older. This rare genetic disease leads to tissue swelling attacks in the body that can be life-threatening. It is caused by a deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system, KalVista Pharmaceuticals shared.

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Fette Compacting India and Goa College of Pharmacy join hands to upskill Goa pharma students

Express Pharma

Fette Compacting India, a global leader in tablet production technology, has signed a Memorandum of Understanding (MoU) with Goa College of Pharmacy to upskill students and prepare them for careers in the pharmaceutical industry. The agreement was signed at Goa College of Pharmacy’s campus in Panaji by Ashok Gourish, Managing Director of Fette Compacting India, and Dr Gopalkrishna Rao, Principal of Goa College of Pharmacy.

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Osivax publishes phase 2a results supporting dual flu vaccine approach

Pharma Times

Subscribe Advertise About us Follow Follow Follow Follow GO Magazine PharmaTimes Magazine Archive Web Exclusives News Competitions Appointments Business Insights Webinars Thought Leadership Osivax publishes phase 2a results supporting dual flu vaccine approach by John Pinching | 10th Jul 2025 | News OVX836 shown to be safe and effective alongside seasonal Fluarix Tetra Osivax has released phase 2a trial data confirming that its broad-spectrum flu vaccine candidate, OVX836, can be safely co-admin

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STAT+: California stem cell agency shutting down unique human tissue biobank

STAT

The California Institute for Regenerative Medicine has for a decade owned a human stem cell biobank that was once the largest of its kind in the world. But the agency and its partners are now racing to sell off thousands of precious samples in a massive fire sale before many of them are discarded. Created by state voters to fund stem cell research, the institute amassed a vast array of precursor cells called induced pluripotent stem cells, or iPSCs.

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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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The MEK effect on cancer — a slow and steady approach to drug resistance

PharmaVoice

Skip to main content CONTINUE TO SITE ➞ Dont miss the most important voices in pharma Let PharmaVoices free newsletter keep you informed on what industry leaders are saying, straight from your inbox. By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Voices Q&A Profiles Biotech Spotlight First 90 Days Opinions Topics Biotech Commercialization Leadership Manufacturing Patient Pharma Policy & Regulation Research & Deve

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New research partnerships aim to transform drug development and production

The Pharmacist

A major UK research funding programme will see new collaborations between academia and the pharmaceutical industry aimed at speeding up drug manufacturing, developing next-generation therapies, and tackling environmental challenges in medicine production. Twenty-three new Prosperity Partnerships – jointly funded by UK Research and Innovation’s Engineering and Physical Sciences Research Council (EPSRC), industry, and academic institutions […] The post New research partnerships aim to transf

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Clearsynth receives government backing to advance India’s deuterated chemical production

Express Pharma

Clearsynth Labs has obtained grants from the Technology Development Board (TDB), which is part of the Department of Science and Technology (DST), Government of India, to build a dedicated manufacturing facility for the commercial-scale production of deuterated reagents, intermediates, and building blocks. This is a significant step towards enhancing India’s capabilities in advanced specialty chemicals.

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Patients with ultra-rare diseases worry new FDA rules will leave them without treatment

The Guardian - Pharmaceutical Industry

Testing is difficult for drugs for rare diseases, and new rules may make it harder for sufferers to obtain life-saving drugs US drug regulators have increasingly signaled a focus on faster approvals and rare diseases, but patients with ultra-rare ailments fear they are falling through the cracks, especially given challenges to conducting clinical trials.

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Why Every Small Business Needs an HCM Solution: A Comprehensive Guide

Managing HR tasks like payroll, compliance, and employee data can overwhelm small businesses. That’s where a Human Capital Management (HCM) solution comes in. Our eBook, Why Every Small Business Needs an HCM Solution: A Comprehensive Guide , shows how an HCM system automates tedious processes, ensuring your business stays compliant and efficient. You’ll learn how to simplify payroll, eliminate costly errors, and empower your employees with self-service tools.

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STAT+: Cogent Biosciences drug improves symptoms of chronic immune disorder, study results show

STAT

Cogent Biosciences said Monday that its experimental drug reduced the symptoms of a chronic immune disorder called indolent systemic mastocytosis. The results mean the drug achieved the goals of a Phase 3 study, but a comparison to a rival treatment from Blueprint Medicines remains muddled.  In its study, Cogent’s drug, called bezuclastinib, showed a 24-point improvement in a patient-reported symptoms score, compared to a 15-point improvement for participants given a placebo.

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GLP-1 roadblocks: Discontinuation rates soar as insurers clamp down

PharmaVoice

Skip to main content CONTINUE TO SITE ➞ Dont miss the most important voices in pharma Let PharmaVoices free newsletter keep you informed on what industry leaders are saying, straight from your inbox. By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Voices Q&A Profiles Biotech Spotlight First 90 Days Opinions Topics Biotech Commercialization Leadership Manufacturing Patient Pharma Policy & Regulation Research & Deve

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Boehringer raises hope of oral drug for diabetic eye disease

pharmaphorum

Boehringer has started a phase 2 trial of an oral therapy that could help people with diabetic macular oedema avoid regular injections into the eye

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Pulmonary arterial hypertension market to reach $9.3 bn in 7MM by 2034

Express Pharma

The pulmonary arterial hypertension (PAH) market across the seven major markets (7MM*) is poised to grow at a compound annual growth rate (CAGR) of 2.0 per cent from $7.6 billion in 2024 to $9.3 billion in 2034, driven by the adoption of triple combination therapy, novel prostacyclin formulations, and the entry of disease-modifying therapies, according to GlobalData.

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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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ASBM Announces Retirement of Doug Badger,Welcomes Beato and Downing to Board

Safe Biologics

ARLINGTON, VA — July 7, 2025 — The Alliance for Safe Biologic Medicines (ASBM) today announced the retirement of longtime Board member Doug Badger, and the appointment of two new members to its Board of Directors: Cristina V. Beato, MD, and Chris Downing, both of whom previously served in senior leadership roles at the U.S. Department of Health and Services (HHS) from 2001 to 2008.

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U.S. sees most single-year measles cases since it declared disease eliminated

STAT

The United States has now recorded more confirmed measles cases this year than in any year in well over a quarter-century — and 2025 is only just past the halfway mark. On Wednesday, the Centers for Disease Control and Prevention reported that the number of confirmed measles cases in the country had risen to 1,288, exceeding the 1,274 cases reported in 2019, to become the highest single-year total since the United States declared measles eliminated in 2000.

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Is biopharma dealmaking getting hot again?

PharmaVoice

Skip to main content CONTINUE TO SITE ➞ Dont miss the most important voices in pharma Let PharmaVoices free newsletter keep you informed on what industry leaders are saying, straight from your inbox. By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Voices Q&A Profiles Biotech Spotlight First 90 Days Opinions Topics Biotech Commercialization Leadership Manufacturing Patient Pharma Policy & Regulation Research & Deve

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VarmX secures US approval to begin phase 3 trial of urgent surgery treatment

Pharma Times

Subscribe Advertise About us Follow Follow Follow Follow GO Magazine PharmaTimes Magazine Archive Web Exclusives News Competitions Appointments Business Insights Webinars Thought Leadership VarmX secures US approval to begin phase 3 trial of urgent surgery treatment by John Pinching | 8th Jul 2025 | News New trial to test VMX-C001’s effectiveness in restoring coagulation in FXa DOAC patients Biotech company VarmX has received clearance from the US FDA to begin a phase 3 trial of its lead candida

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Best Practices to Streamline Compensation Management: A Foundation for Growth

Speaker: Joe Sharpe and James Carlson

Payroll optimization can be one of the most time-consuming and complex factors of small business management. Yet, organizations that crack the code on streamlining employee compensation often discover innovative avenues for growth. With the right strategies in place, outsourcing and streamlining payroll processes can result in substantial time and resource savings.

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CDC’s vaccine advisory committee overhaul raises concerns for vaccine oversight: GlobalData

Express Pharma

On June 9, 2025, in a significant and controversial shake-up, US Secretary of Health and Human Services Robert F. Kennedy Jr. dismissed all 17 members of the Centers for Disease Control and Prevention (CDC) Advisory Committee for Immunization Practices (ACIP), the body responsible for advising the federal government on vaccine safety. This decision has raised alarms among public health experts and stakeholders in the vaccine development community, says GlobalData.

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Episode 1042: Early Norepinephrine Use in Septic Shock is Associated with Reduced Mortality

Pharmacy Joe

In this episode, I’ll discuss early vs delayed norepinephrine use in patients with septic shock. Episode 1042: Early Norepinephrine Use in Septic Shock is Associated with Reduced Mortality Subscribe on iTunes , Android , or Stitcher The optimal timing of when to start a vasopressor in patients with septic shock is unknown. While early norepinephrine makes intuitive sense, there are many reasons why a clinician may wish to see if a patient first responds to IV fluids – including that vasopr

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Getting off antidepressants isn’t as risky as is widely believed, review suggests

STAT

Antidepressant use has been increasing for decades in the U.S., particularly among women and young people. And with more patients starting the drugs, there have been growing concerns about how to get off of them. Stopping, it’s widely believed, can come with an array of harmful side effects. “I know people, including members of my family, who’ve had a much worse time getting off of SSRIs than they have getting off of heroin,” health secretary Robert F.

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Generative AI Can Design Drugs. But Can It Own Them?

Drug Patent Watch

"The Future of Pharma is in Flux: Can AI-Designed Medicines Be Owned? As the pharmaceutical industry grapples with the rise of generative AI, a new question is emerging: who owns the rights to medicines designed by machines? In a groundbreaking development, researchers have successfully used AI to design novel compounds with unprecedented efficacy.

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Enhance Healthcare Efficiency With Top Payroll & HCM Services

Running a healthcare facility requires precision and care, not just for patients but also for your staff. Our guide, "A Buyer’s Guide to Payroll & HCM Services," helps healthcare providers choose the best provider. Efficient payroll management ensures timely, accurate payments, critical for maintaining staff morale and trust. Compliance support helps navigate complex healthcare regulations and avoid costly fines.

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Samsung builds digital health credentials with Xealth buy

pharmaphorum

Samsung has reached a deal to buy Xealth, a US company set up to help healthcare professionals prescribe digital health technologies.

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Pharmexcil urges balance between compliance and commerce to safeguard India’s export momentum and ‘Pharmacy of the World’ status

Express Pharma

The Pharmaceuticals Export Promotion Council of India (Pharmexcil) has expressed urgent concerns over the recent circular issued by the Central Drugs Standard Control Organization (CDSCO), which mandates exclusive online submission of Certificate of Pharmaceutical Product (COPP) applications via the ONDLS portal. While the Council supports digital integration and modernization of regulatory frameworks, it foresees a major disruption to India’s pharmaceutical exports – particularly to Rest

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Actithera closes $75.5m Series A to advance radioligand therapy platform

Pharma Times

Subscribe Advertise About us Follow Follow Follow Follow GO Magazine PharmaTimes Magazine Archive Web Exclusives News Competitions Appointments Business Insights Webinars Thought Leadership Actithera closes $75.5m Series A to advance radioligand therapy platform by John Pinching | 9th Jul 2025 | News Funding to support lead candidate targeting FAP and pipeline expansion Actithera has secured $75.5 million in an oversubscribed Series A round to support the clinical development of its lead radioli

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Opinion: ER docs are too quick to assume cannabis users are experiencing this rare side effect

STAT

Human health is complex and diagnostic tools and their limitations, so missing a diagnosis is sometimes unavoidable. But every missed diagnosis has the potential to cause harm to a patient, as is demonstrated by a dangerous trend I’ve noticed within emergency departments: a tendency to attribute symptoms too readily to cannabis hyperemesis syndrome (CHS) without adequate diagnostic work-up.

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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.