Bath, England – When you’re an autistic teenager living with chronic pain, getting treatment for your pain can be a challenging experience. That’s according to a group of young people who’ve spoken to Dr. Abbie Jordan of the Department of Psychology and Centre for Pain Research at The University of...
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Adding enasidenib to azacitidine significantly improved responses in older patients with newly diagnosed, IDH2-mutant acute myeloid leukemia (AML), according to research published in The Lancet Oncology. In a phase 1b/2 trial (ClinicalTrials.gov Identifier: NCT02677922), researchers compared enasidenib plus azacitidine with azacitidine monotherapy. The trial enrolled 107 patients with newly diagnosed, IDH2-mutant AML who were...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today announced the approval of its Clinical Trial Authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for the EXPEDITION Study of...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome. Individuals with Dravet syndrome, the most common...
The foundation End AxD, a nonprofit dedicated to research, treatment and a cure for Alexander disease, has donated $74,000 to UMass Chan Medical School to explore a gene therapy treatment for the fatal neurological disease. The funding will go to Jun Xie, PhD, associate professor of microbiology & physiological systems,...
SAN DIEGO – Endeavor BioMedicines, a clinical-stage precision medicine company targeting the core drivers of multiple terminal diseases including oncology and fibrosis, announced that the company has dosed its first patient in a Phase 2 trial of taladegib (ENV-101) to treat idiopathic pulmonary fibrosis (IPF). Taladegib is a small-molecule inhibitor...
SAN DIEGO, Calif. — Endeavor BioMedicines, a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases, today announced successful completion of its Phase 2a clinical trial evaluating the company’s lead investigational compound, ENV-101, in patients with idiopathic pulmonary fibrosis (IPF). ENV-101...
New York, NY – Endometrial cancer — which develops in the lining of the uterus (womb) and is sometimes called uterine cancer — is on the rise in the U.S. In 1987, there were 35,000 cases annually. That number has nearly doubled in 2023 to more than 66,000 cases. Deaths from...
DURHAM, N.C. — A team of physicians, neuroscientists and engineers at Duke University has demonstrated two new strategies that use deep brain stimulation to improve the symptoms of Parkinson’s disease. By simultaneously targeting two key brain structures and using a novel self-adjusting device, the team showed that they can efficiently...
PTC Therapeutics’ Upstaza, a gene therapy for an ultra-rare genetic disorder and the first to be directly infused in the brain, is one step closer to reaching patients in the U.K. The National Institute for Health and Care Excellence (NICE) has recommended the therapy to treat patients with aromatic L-amino acid...