A team led by a University of Florida Health scientist last month landed what it believes is the first National Institutes of Health grant ever awarded specifically to study a rare neurological disease that affects the lives of children and their families.
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GAINESVILLE — A dog born with a deadly disease that prevents the body from using stored sugar has survived 20 months and is still healthy after receiving gene therapy at the University of Florida — putting scientists a step closer to finding a cure for the disorder in children. Called...
Houston , TX — University of Houston Assistant Professor of Biology and Biochemistry Quentin Vicens has been awarded a $1.2 million grant from the National Institute of General Medical Sciences to unravel the mystery of Z-RNA – an enigmatic RNA structure within our cells that plays a critical role in...
PARIS – GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that its gene therapy LUMEVOQ® has been granted Promising Innovative Medicine (PIM) designation by the UK’s Medicines and...
The son of Vitaliy Svichynskyi and Maria Svichynska was nearly two months old when he suddenly started to lose the ability to move. The family soon learned their baby boy Dmytro was born with Spinal Muscular Atrophy (SMA) Type 1, the most severe type of the rare genetic disorder found in...
Newark — Ultima Genomics, Inc. and Genome Insight, Inc. have signed an agreement to collaborate to bring affordable whole genome sequencing solutions to cancer patients. As part of the agreement, Genome Insight will join the early access program for Ultima’s high-throughput NGS instrument platform, the UG 100™, and develop an optimized...
Ultimovacs has announced that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) for the company’s universal cancer vaccine UV1 for the treatment of stage IIB – IV melanoma. UV1, as an add-on therapy to checkpoint inhibitors ipilimumab and nivolumab, is currently being studied as a first-line...
Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). If authorised, Ultomiris would be the first and only approved long-acting C5 complement inhibitor for the treatment of...
WILMINGTON, Del. — ULTOMIRIS® (ravulizumab-cwvz) has been approved in the United States as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The approval by the US Food and Drug Administration (FDA) was based on...
NOVATO, Calif., VANCOUVER, British Columbia and LONDON, England — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO) today announced interim data from the Phase 2 portion of the Phase 2/3 Orbit study demonstrating that treatment with setrusumab (UX143) significantly reduced incidence of fractures in patients...