Columbia University

The FDA approved Alyftrek, a once-daily oral combination for cystic fibrosis in adults and children aged 6 and older. It is Vertex Pharmaceuticals' fifth CFTR modulator, offering efficacy in 31 additional mutations and lower sweat chloride levels than TRIKAFTA. The approval is based on a comprehensive Phase 3 program involving over 1,000 patients globally, demonstrating non-inferiority to TRIKAFTA and significant improvements in sweat chloride levels.

The FDA approved Vertex Pharmaceuticals' once-daily oral combination of vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) for cystic fibrosis in adults and children 6 years and up. The drug targets additional CFTR gene mutations, expanding eligibility to 150 U.S. patients. It demonstrated non-inferiority to Trikafta in phase III trials, showing significant improvements in lung function and sweat chloride levels. Common side effects include cough, nasopharyngitis, and increased liver enzymes. The drug carries a boxed warning for liver injury and failure.

The FDA approved vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) for cystic fibrosis in patients 6+ with at least 1 F508del mutation or a responsive mutation. Alyftrek, a once-daily CFTR modulator, met primary and secondary endpoints in phase 3 trials, showing non-inferiority to Trikafta in ppFEV1 and superiority in reducing sweat chloride levels.

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.

Scott A. Small agrees with most of Dennis J. Selkoe's proposals on amyloid-clearing drugs but questions if extracellular amyloid aggregates are a cause of AD, suggesting they are a defining feature with cognitive detriment. He argues intracellular pathology, like endosomal enlargement, may drive AD's neurodegenerative process, necessitating clinical trials to resolve the debate on AD's prime driver.

FDA approves expanded use of Trikafta for cystic fibrosis patients aged 2+ with at least 1 F508del mutation or responsive mutation, benefiting ~300 more US patients. Trikafta, an oral medication, enhances CFTR protein function, improving lung function and quality of life. Vertex also secures approval for Alyftek, a once-daily CFTR modulator for patients 6+.

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.

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.