London, UK – Bowel cancer cells have the ability to regulate their growth using a genetic on-off switch to maximise their chances of survival, a phenomenon that’s been observed for the first time by researchers at UCL and University Medical Center Utrecht. The number of genetic mutations in a cancer...
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Lancaster, England – The genetic disease Huntington’s not only affects nerve cells in the brain but also has widespread effects on microscopic blood vessels according to research. These changes to the vasculature were also observed in the pre-symptomatic stages of the disease, demonstrating the potential for this research for predicting...
Tel Aviv, Israel – In a breakthrough study an international team of scientists led by researchers from Tel Aviv and Glasgow Universities, the ‘cat parasite’ Toxoplasma gondii was engineered to deliver drugs to the human brain. The study was led by Prof. Oded Rechavi from the Department of Neurobiology and...
Hinxton, England – For the first time, scientists have systematically analysed somatic mutations in stomach lining tissue to unpick mutational processes, some of which can lead to cancer. The team also uncovered hints of a potential new cause of stomach cancer that needs further research. Researchers at the Wellcome Sanger...
Nashville, Tennessee – Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD). The therapy, nonmyeloablative haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy), is proving to have equivalent efficacy...
Amanda Hamacher was in grade school when she began to lose her sight and hearing as a result of a rare genetic disorder called Alstrom syndrome. Now, the 21-year-old cannot see and has severe hearing loss. Yet, she enjoys a newfound sense of freedom thanks to a program sponsored by...
Intestinal permeability and an overgrowth of bacteria in the small intestine are both associated with nonalcoholic fatty liver disease (NAFLD). These findings are revealed in a new study in the June issue of Hepatology, a journal published by John Wiley & Sons on behalf of the American Association for the...
AUSTIN, TX – IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients...
Hello everyone! My name is Claire Bevec and I’m so excited to announce my monthly column that I will be writing for Harmony 4 Hope. I am so thankful for this opportunity to share some of the most beautiful stories about life with a rare disease. Each month I will...
Daix (France) and New York, NY – Inventiva (Euronext Paris and Nasdaq: IVA) (“Inventiva” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of oral therapies for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), today announces the publication in Journal of Hepatology Reports, a peer-reviewed, scientific journal, of results...