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...
treatment News
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Inc., today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England.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. – 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...
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...
The increasing amount of patients with obesity and type 2 diabetes benefit greatly from the recently developed GIPR:GLP-1R co-agonists. These novel compounds lead to substantial weight loss, offering a revolutionary approach to patients worldwide. Although the hormone glucose-dependent insulinotropic polypeptide (GIP) was already shown by Helmholtz Munich scientists to decrease...
MINNEAPOLIS – OX2 Therapeutics, Inc., a privately held Minneapolis based company, announced today encouraging early results from phase 1 human trials of their new cancer treatment. Developed to combat recurrent high-grade brain tumors, the treatment utilizes a newly developed peptide inhibitor. “OX2 works to explore the therapeutic potential of the new immune...