NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor...
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Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food...
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...
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Inc., today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England.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...
NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. today announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder. The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with...
NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has resubmitted its Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) to the U.S. Food and Drug Administration (FDA or...
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...