YALE UNIVERSITY

AAP leader Sanjay Singh urges donations for Prashant and Neha Yadav, parents of a 10-month-old with SMA, needing ₹10 crore for Zolgensma. They've raised ₹1.5 crore. Singh advocates for state intervention and donates ₹1 lakh. Zolgensma costs $1.7 million, while Risdiplam could be ₹3,000 if freed from patents.

AACR focuses on cancer prevention and early detection, with Kornelia Polyak's pioneering research highlighting the importance of early-stage disease study. The AACR established the Cancer Prevention Working Group in 2021 to support innovative science and public education. Liquid biopsies and multi-cancer early detection (MCED) tests are being developed to improve screening. The NCI's National Cancer Plan and President Biden's proclamation of April 2024 as National Cancer Prevention and Early Detection Month emphasize the urgency of these efforts. Researchers are exploring liquid biopsies, MCED, cancer interception, and population science to advance early detection and prevention strategies.

A new schizophrenia drug, Cobenfy, showed improvement in patients with few side effects, but high dropout rates typical in such studies. Cobenfy, approved in September, works differently in the brain and includes trospium to mitigate side effects like nausea. Despite the high dropout rates, some patients experienced meaningful symptom improvement, and the drug led to weight loss. The cost of Cobenfy is significantly higher than generic alternatives, raising concerns about insurance coverage.

Yale University invites investigators and clinical trial staff to a Good Clinical Practice (GCP) refresher training on Nov. 13, 11 a.m.-2 p.m. in hybrid format at Cohen Auditorium or via Zoom. Lunch and refreshments provided for in-person attendees. Register at [link].

Mona Shahriari, MD, FAAD, discussed itch, psoriasis, skin of color, and future dermatology at the Fall Clinical Dermatology Conference. Her sessions included 'Making the Connection Between Prurigo Nodularis, Atopic Dermatitis, and Itch' and 'Practical Insights and Pitfalls for Psoriatic Disease in Skin of Color'. She emphasized the importance of understanding itch's impact on patients' lives and the need for collaborative care in treating psoriatic disease in skin of color.

A Yale University study identified proteins PRDM1 and SGK1 over-expressed in regulatory T-cells, causing dysfunction in MS. Suhayl Dhib-Jalbut, MD, believes targeting these proteins could lead to new MS treatments, though drug development is a lengthy process.

A pig kidney preserved at subzero temperatures was successfully transported across the Atlantic multiple times, showcasing a novel technology that extends organ preservation and transportation, potentially increasing transplants and saving lives. The kidneys were preserved in X-Therma's XT-ViVo® solution in X-Therma's TimeSeal® device, which uses peptoids to prevent ice crystal formation, enabling ice-free preservation at 0°C to -20°C. The FDA granted Breakthrough Device status to XT-ViVo® and TimeSeal® for human kidney preservation up to 120 hours.

Researchers used AI to design new DNA switches, called synthetic cis-regulatory elements (CREs), that precisely control gene expression in specific cell types, outperforming naturally occurring CREs in cell-type specificity.

Pig kidneys preserved at subzero temperatures were successfully transported across the Atlantic multiple times using X-Therma's FDA Breakthrough-designated technology, demonstrating potential to extend organ storage and enable long-distance transportation.

Methadone found more effective than buprenorphine/naloxone in keeping patients in opioid use disorder treatment, with lower discontinuation rates. Methadone's effectiveness persisted despite fentanyl's rise. Regulatory changes now allow for telehealth methadone prescriptions, potentially improving patient retention.