Columbia University

ALYFTREK™, a once-daily CFTR modulator, approved by FDA for cystic fibrosis patients aged 6+ with specific mutations, shows non-inferiority to TRIKAFTA® in clinical trials, offering a new treatment option with improved efficacy and convenience.

Sexism linked to faster memory decline in U.S. women, study finds. Women born in more sexist states experience memory decline similar to women 9 years older. Addressing social inequities may help lower Alzheimer’s burden among women.

ALYFTREK, a once-daily CFTR modulator, received FDA approval for cystic fibrosis, treating 31 additional mutations and offering lower sweat chloride levels than TRIKAFTA. It was tested in a comprehensive Phase 3 program involving over 1,000 patients globally, demonstrating efficacy and safety. ALYFTREK aims to improve patient care with convenient dosing and expanded mutation coverage.

A team of physicians and engineers, including ENT surgeon Anil Lalwani and mechanical engineer Jeffrey Kysar, have developed an ultrasharp and ultrathin microneedle designed for precision medicine in the inner ear. The microneedle aims to deliver gene therapies and diagnose inner ear disorders like Meniere’s disease without damaging the cochlea's delicate membrane. The device, created through years of collaboration and enabled by 3D printing technology, has shown promising results in animal studies and is being commercialized by Haystack Medical for potential human use.

Dr. Gary Gibson hypothesizes that thiamine deficiency alters glucose metabolism in brain cells, leading to Alzheimer's. His research led to a nationwide clinical trial evaluating benfotiamine, an oral drug boosting thiamine levels, as a potential treatment.

Seth Lederman, co-founder, CEO & Chairman of Tonix Pharmaceuticals, is a physician, scientist, and entrepreneur with extensive experience in molecular immunology and autoimmune diseases. Steve Gardner, CEO of Precision Life, has over 30 years of experience in precision medicine, drug discovery, and computational biology, and is involved in genomics and AI-enabled drug discovery.

Clene received FDA guidance on an accelerated approval pathway for CNM-Au8 in ALS, recommending leveraging additional NfL data from EAPs and the HEALEY ALS Platform Trial. Clene plans to submit an NDA in mid-2025, incorporating EAP NfL biomarker analyses, and commence the RESTORE-ALS Phase 3 trial before NDA submission.

The murder of UnitedHealth CEO Brian Thompson by Luigi Mangione has sparked widespread reactions, highlighting deep frustrations with the U.S. healthcare system. Many, including those with good insurance, face crippling medical debt and systemic issues, fueling anger towards healthcare executives profiting from a broken system.