ASSIUT, Egypt – Seven previously unreported mutations linked to type 1 Gaucher disease were identified in a study analyzing the genetic code of dozens of people with the condition in Egypt. “All patients carrying these novel variants exhibited reduced enzyme levels and clinical symptoms characteristic of type 1 [Gaucher disease], without...
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PALO ALTO, Calif. — Eiger BioPharmaceuticals, Inc, a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases, today announced that it and its partner AnGes, Inc. received marketing approval from the Ministry of Health, Labour and Welfare for Zokinvy (lonafarnib), a treatment for Hutchinson-Gilford progeria...
The investigational oral selective estrogen receptor degrader (SERD) elacestrant significantly decreased the risk of death or disease progression and increased progression-free survival compared with standard-of-care endocrine therapy for postmenopausal patients with estrogen receptor (ER)-positive/HER2-negative metastatic breast cancers that progressed on prior endocrine and targeted therapies, according to results from the...
BOSTON, Mass. — Elicio Therapeutics, Inc., a clinical-stage biotechnology company developing a pipeline of novel immunotherapies for the treatment of cancer, today announced that the first patient has been dosed at Northwell Health Cancer Institute and the Feinstein Institutes for Medical Research, New York, as part of the randomized Phase...
Blacksburg, VA – Every 65 seconds, someone in the United States develops Alzheimer’s disease, a devastating form of dementia that affects 6.2 million Americans. Though it was initially identified almost 120 years ago, Alzheimer’s disease is a progressive neurological disorder with no cure and few treatments. It starts out with minor...
LAS VEGAS — Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either...
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...
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,...
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...