Mirum Pharmaceuticals recently made headlines as they secured FDA approval for Ctexli, also known as chenodiol, as the first treatment for cerebrotendinous xanthomatosis (CTX). CTX, a rare genetic lipid storage disease, affects an estimated 3 to 5 individuals per 100,000. This approval came after a
Key players in the pharmaceutical landscape, Summit Therapeutics and Pfizer, are embarking on a noteworthy collaboration to test out Summit’s experimental immunotherapy, ivonescimab, combined with Pfizer’s sophisticated antibody-drug conjugates (ADCs) in upcoming clinical trials targeting specific
Appendiceal cancer, a rare malignancy, presents significant challenges due to the lack of standardized screening guidelines, identifiable risk factors, and a common tumor classification system. These hurdles often result in delayed diagnoses and poor prognoses for patients. The estimated annual
Human aging is a complex process influenced by a combination of genetic and environmental factors. While the role of genetics has been widely studied, recent research highlights the significant impact of environmental exposures on aging and premature mortality. This article delves into the findings
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder that progressively deteriorates muscle function, posing a major challenge for patients and their families. Recently, a promising breakthrough has emerged in the form of Solid Biosciences' experimental gene therapy, SGT-003, which
In a groundbreaking study published in JAMA, researchers from Mass General Brigham have demonstrated the potential of artificial intelligence (AI) to revolutionize patient screening for clinical trials. The study, led by co-senior authors Samuel (Sandy) Aronson and Alexander Blood, focused on a
