MASSACHUSETTS GENERAL HOSPITAL

Pharmaust rebrands as Neurizon Therapeutics, focusing on neuro-degenerative diseases like ALS, using repurposed animal drug monepantel. The company plans a pivotal phase II/III trial with FDA fast-track approval potential, supported by a $900,000 grant from Fight MND.

Members of the US Pancreatic Disease Study Group include C. Mel Wilcox, Ji Young Bang, Akwi Asombang, Chloe Bennett, Yan Bi, Wojciech Blogowski, James Buxbaum, Wei-Shen Chin, Darwin Conwell, Gregory A. Coté, Timothy B. Gardner, Pramod Garg, Nalini Guda, Robert Hawes, Yasmin G. Hernandez-Barco, Emily Jonica, Prashant Kedia, Thomas Kowalski, Vivek Kumbhari, Linda Lee, Jorge Machicado, Desiree Morgan, Thiruvengadam Muniraj, Udayakumar Navaneethan, Veeral Oza, Swati Pawa, Rajesh Puri, Amit Rastogi, D. Nageshwar Reddy, Monica Saumoy, Mandeep Sawhney, Santhi Swaroop Vege, Rupjyoti Talukdar, Paul Tarnasky, Felix Tellez-Avila, Shyam Thakkar, Nikhil Thiruvengadam, Elaina Vivian, Irving Waxman, Field F. Willingham, Shyam Varadarajulu.

STAT's First Opinion platform publishes letters in response to essays, fostering discussion. Regina LaRocque disagrees with 'institutional neutrality,' quoting Elie Wiesel. Gerald Keusch praises mRNA vaccine development's scientific foundation. Terry Wilcox advocates for the FDA's Rare Disease Innovation Hub to ensure rare disease patients benefit from advancements.

Meiler lab awarded $46 million to develop a 'super-vaccine' against alphaviruses, leveraging computational techniques and a multidisciplinary team.

FDA approved Shield blood test in July as primary screening for average-risk colorectal cancer, detecting cancers in 83% of participants but only 13% of precancerous polyps. Colonoscopy remains the gold standard, with experts uncertain about Shield's role in current screening options. Key questions include test frequency, cost, and impact on cancer deaths.

Most boys treated with eli-cel gene therapy for cerebral adrenoleukodystrophy (CALD) are faring well six years later, with 94% showing no neurological decline and 80% free of disability. However, six of 35 patients developed blood cancers linked to the therapy, highlighting the risks associated with gene therapies.

Resilience training must be based on natural development, with a therapeutic alliance crucial for success. Misperceived resilience, often due to stoicism or pressure, can mask ongoing issues, risking inadequate treatment. Clinicians must remain vigilant against pressures that may lead to false perceptions of resilience, ensuring proper care.

Thirty years after the discovery of microRNAs, which regulate gene activity, the Nobel Prize in Physiology or Medicine was awarded to their discoverers. Despite potential applications in treating cancer and heart disease, no microRNA-based drugs have been approved by the FDA. Early clinical trials faced challenges with delivery and immune response, but advancements in technology and increased interest from the Nobel Prize may drive future success in developing microRNA-based medicines.

DelveInsight's Menopause Market Insights, Epidemiology, and Market Forecast-2034 report analyzes market trends, epidemiology, and emerging therapies in the US, EU4, UK, and Japan. Key companies include GlaxoSmithKline, Pfizer, Bayer, and others. The market size was ~USD 10,968 million in 2022, with hormonal therapy dominating but expected to decline as non-hormonal therapies gain prevalence. Notable therapies include VEOZAH, a non-hormonal treatment approved by the FDA in 2023.