Toyoake, Japan – Over the past decades, scientists have made substantial progress unveiling the underlying mechanisms behind many psychiatric disorders. Every year, new genetic mutations or protein dysregulations are identified as potential culprits for the symptoms, and sometimes even the root causes of complex neurological diseases, including autism spectrum disorder...
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The increasing amount of patients with obesity and type 2 diabetes benefit greatly from the recently developed GIPR:GLP-1R co-agonists. These novel compounds lead to substantial weight loss, offering a revolutionary approach to patients worldwide. Although the hormone glucose-dependent insulinotropic polypeptide (GIP) was already shown by Helmholtz Munich scientists to decrease...
South Bend, Indiana – Glioblastoma is one of the most treatment-resistant cancers, with those diagnosed surviving for less than two years. In a new study in NPJ Genomic Medicine, researchers at the University of Notre Dame have found that a largely understudied cell could offer new insight into how the aggressive,...
A close relative of the microorganism that causes tuberculosis in humans has been found to form spores. This is a sensational finding because researchers have long been convinced that these kinds of bacteria–the mycobacteria–were incapable of forming spores. Leif Kirsebom’s research group at Uppsala University now has photographic proof, obtained...
Houston, Texas – In a report published in Nature Cell Biology, researchers at Baylor College of Medicine, Texas Children’s Hospital, the University of Manitoba and collaborating institutions revealed an unexpected way in which the protein OTX2 drives the progression of medulloblastoma – the most common aggressive childhood brain cancer. The...
MINNEAPOLIS – OX2 Therapeutics, Inc., a privately held Minneapolis based company, announced today encouraging early results from phase 1 human trials of their new cancer treatment. Developed to combat recurrent high-grade brain tumors, the treatment utilizes a newly developed peptide inhibitor. “OX2 works to explore the therapeutic potential of the new immune...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for...
Nijmegen, The Netherlands – TB is a major threat to public health, being among the leading causes of death worldwide. In 2021, the disease claimed the lives of 1.6 million people, making it the second leading infectious killer after COVID-19.[1] Drug-resistant TB and long treatment regimens have increased the urgency...
NEW YORK, NY — Parent Project Muscular Dystrophy (PPMD) and the World Duchenne Organization (WDO), along with WDO’s members and affiliates, proudly herald the United Nations marking a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day (WDAD), set to be...
Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In addition, a number of policies implemented in response to COVID-19 are expected to have...