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Inc., today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England.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...
treatment News
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. — Today Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301-treated patients (n=18) demonstrated a statistically...
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...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease, a rare, inherited neurodegenerative disorder...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for...