Pittsburgh, PA – In a Nature Metabolism paper published today, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, life-threatening and currently incurable genetic disease with devastating consequences. Barth syndrome affects about 1 in every 300,000 to...
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Utrecht, Netherlands – The Organoid Group (Hubrecht Institute) and the Rare Cancers Genomics Team (IARC/WHO) found a way to grow samples of different types of neuroendocrine tumors (NETs) in the lab. While generating their new model, the researchers discovered that some pulmonary NETs need the protein EGF to be able...
NASHVILLE, Tenn. – A potential schizophrenia treatment discovered through the Warren Center for Neuroscience Drug Discovery has been cleared by the U.S. Food and Drug Administration for use in phase 1 clinical trials—the third WCNDD therapeutic to reach that benchmark. The drug is an allosteric modulator that works through a...
BIRMINGHAM, Ala. – It has been uncertain how Mycobacterium tuberculosis deflects the immune response in humans, though evidence has pointed to host immunometabolism — the intrinsic link between metabolism in immune cells and their immune function. The pathogen M. tuberculosis is known to disrupt a metabolic pathway called glycolysis in...
Treating children with inflammatory bowel disease (IBD) usually involves the same steroids-based medication prescribed to adults. But such treatments can have negative side effects for kids and teens dealing with IBD. Dr. Raanan Shamir of Tel Aviv University’s Sackler School of Medicine and Schneider Children’s Medical Centre shows that there...
LYON, France – POXEL SA (Euronext – POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders, today announced a new strategic direction to focus its pipeline on high value, rare metabolic indications and NASH, with...
LYON, France – POXEL SA (Euronext: POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders, today announced that Elizabeth Woo has joined the Company as Senior Vice President, Investor Relations, Public Relations and Corporate Communications,...
Washington, DC – PPMD has provided $250,000 in funding to Rare Disease Research, LLC (RDR), to open a second independent rare disease clinical research site in the Raleigh-Durham metropolitan area in North Carolina. With this funding commitment, we aim to support RDR’s creation of an avenue that increases patient access...
South Bend, Indiana – Adding a pre-ketone supplement — a component of a high-fat, low-carb ketogenic diet — to a type of cancer therapy in a laboratory setting was highly effective for treating prostate cancer, researchers from the University of Notre Dame found. Recently published online in the journal Cancer...
GERMANTOWN, Md. – Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced that the first patient has been dosed in the Phase I study of PRGN-2012, a first-in-class, investigational off-the-shelf (OTS) AdenoVerse™ immunotherapy in adult patients with recurrent respiratory...