Washington DC — FDA announced a pilot program, Support for clinical Trials Advancing Rare disease Therapeutics (START), intended to support development of novel drug and biological products for rare diseases. The agency is establishing a pilot program for a limited number of sponsors, intended to allow for more regular communication...
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SOUTH SAN FRANCISCO, Calif. — Genentech, Inc. a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has accepted, under Priority Review, the company’s supplemental Biologics License Application (sBLA) for Xolair® (omalizumab) for the reduction of allergic reactions, including anaphylaxis, that may occur with...
New York, NY – An iron-binding drug that is already approved for treatment of other diseases could provide a novel way to attack ovarian tumors, according to a new study led by Weill Cornell Medicine researchers. The preclinical study, which combined the analysis of human ovarian tumors and animal models...
Creating a strategy to discontinue eculizumab in patients with atypical hemolytic uremic syndrome (aHUS) is reasonable and safe and improves quality of life, according to research in Blood. The strategy should be based on complement genetics. Eculizumab has drastically improved outcomes for patients with aHUS. There’s no clearly defined optimal duration...
Hong Kong – A new study finds that very few patients diagnosed with type 2 diabetes are able to achieve normal blood glucose levels through weight loss alone. A team led by Andrea Luk of the Chinese University of Hong Kong, reported these findings in the open access journal PLOS Medicine....
Greenwich, CT – The Fibrolamellar Cancer Foundation is pleased to announce that it has funded a new study directed by Roshni Dasgupta, MD of the Cincinnati Children’s Hospital Medical Center (CCHMC). Dr. Dasgupta is a leader in surgical oncology and the chair of the Pediatric Surgical Oncology Research Collaborative (PSORC)....
Port Elizabeth, South Africa – Fibrous dysplasia is a benign tumour-like condition in which normal bone is replaced by structurally poor fibro-osseous lamellar bone. It occurs as a result of an early embryonic postzygotic activating mutation of the GNAS gene which manifests clinically in a wide spectrum of disease. Fibrous...
COLUMBUS, Ohio – Scientists are focusing on a new concept in fighting airborne pathogens by manipulating what is called the “switching time,” the point at which a highly regulated immune response gives way to powerful cells that specialize in fighting a specific invading bug. In the case of tuberculosis, Ohio...
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...
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...