NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A...
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NOVATO, Calif. — Ultragenyx Pharmaceutical Inc., today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for...
AMHERST, Mass. – While we often think of diseases as caused by foreign bodies—bacteria or viruses—there are hundreds of diseases affecting humans that result from errors in cellular production of its proteins. A team of researchers led by the University of Massachusetts Amherst recently leveraged the power of cutting-edge technology,...
AMHERST, Mass. – There is a surprising dearth of research about how breast cancer cells can go dormant, spread and then resurface years or even decades later, according to a new review of in vitro breast cancer studies conducted by researchers at the University of Massachusetts Amherst. “[Our review found...
AMHERST, Mass. – Researchers at the University of Massachusetts Amherst recently released a first-of-its-kind study that focuses on the rare autoimmune disorder aplastic anemia to understand how a subset of cells might be trained to correct the overzealous immune response that can lead to fatal autoimmune disorders. The research, published...
AMHERST, Mass. ⎯ An estimated 6.7 million American adults over the age of 65 have Alzheimer’s disease. One of the major challenges with this condition is that there currently is no established, universally recognized method for predicting its onset, and emerging treatments are considered to be effective only in the...
Worcester, Mass. – A study led by UMass Chan Medical School viral immunologists Liisa Selin, MD, PhD, and Anna Gil, PhD, discovered similarities in immune system dysfunction as a potential biomarker among people living with long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). The research also introduced a novel treatment...
Umeå, Sweden – A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies. This is shown in a new study at Umeå University. Protein expressed by the gene naturally prevents the muscles around the...
CHAPEL HILL, N.C. – Angelman syndrome is a rare genetic disorder caused by mutations in the maternally-inherited UBE3A gene and characterized by poor muscle control, limited speech, epilepsy, and intellectual disabilities. Though there isn’t a cure for the condition, new research at the UNC School of Medicine is setting the...
CHAPEL HILL, N.C. — To grow and spread, tumors need new blood vessels, a process called angiogenesis. One growth factor that causes angiogenesis has been identified – vascular endothelial growth factor or VEGF – and drugs to inhibit VEGF are already in use. But not all tumors respond to the...