In June 2020, cannabidiol hit what has become a familiar hurdle in fragile X research: Like many previous drug candidates, it missed its primary target in clinical testing. Among 109 young people with fragile X syndrome who took the drug and 101 who took a placebo, researchers saw no meaningful difference in a...
research News
Birmingham, United Kingdom – A highly aggressive form of leukaemia which is activated by mutations in signalling molecules is maintained by a web of regulatory proteins downstream of these signals. New research published in Cell Reports shows that a complex network of interacting genes activated by this altered signalling may...
Researchers now believe that ALS, or amyotrophic lateral sclerosis, starts well before its debilitating symptoms appear, a finding that they say could eventually lead to an effective treatment. Protein clumps in cells that show up only after damage has occurred, rather than at the onset of the disease, appear responsible...
Stockholm, Sweden – Blood fat-lowering statins could slow the progression of Alzheimer’s disease, at least for some patients. This is the result of a new study led by Karolinska Institutet published in Alzheimer Research and Therapy. But the researchers are cautious in their interpretations and see the results as a...
MEMPHIS, Tenn. – Fibrolamellar carcinoma (FLC) is a rare liver cancer with a poor prognosis if not addressed early with surgery. The disease is caused by a single genetic mutation that creates a fusion protein. Scientists at St. Jude Children’s Research Hospital and The University of Tennessee Health Science Center...
PARIS, France – Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris. The trial combines phases...
Bethesda, MD — The American Gastroenterological Association (AGA) released a new evidence-based guideline recommending the use of blood and stool-based biomarkers to help manage Crohn’s disease, a type of inflammatory bowel disease (IBD). IBD is estimated to affect 2.74 million people in the U.S. The guideline was published today in...
Barcelona, Spain – Directed by Laura Soucek, an ICREA Research Professor, Director of VHIO’s Experimental Therapeutics Program and head of our Models of Cancer Therapies Group, two decades of research aimed at combating resistance to therapy and cancer regression through inhibiting the MYC oncogene—found deregulated in most, if not all...
SYDNEY, Australia — Immutep Limited (formerly known as Prima Biomed), a clinical-stage biotechnology company developing novel LAG-3 immunotherapies for cancer and autoimmune disease, today announces that the first patient has been enrolled and safely dosed in the INSIGHT-005 Phase I trial. The investigator-initiated study jointly funded with Merck KGaA, Darmstadt,...
Manchester, UK – The first patient in the UK has been treated with a pioneering new investigational gene therapy designed to treat the debilitating genetic disorder Gaucher disease. Experts at Northern Care Alliance NHS Foundation Trust (NCA) say the research study is a major milestone for the inherited condition. They...