In the late 1980s, scientists developed a revolutionary approach to treating acute myeloid leukemia (AML), a type of blood cancer. Called differentiation therapy, it amounted to a bona fide cure for many patients. The treatment works by triggering cells “stuck” with a cancerous identity to keep developing and maturing, giving...
research News
Diagnosing von Willebrand disease (VWD) in children younger than 2 is challenging, and data on this population is limited. However, recent research published in Blood Advances has found that infants and toddlers with VWD often have a family history of the disease and experience bleeding within the first year of...
WILMINGTON, Mass. — Charles River Laboratories International, Inc. (NYSE: CRL) today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration with Axovia Therapeutics Ltd. Charles River will manufacture High Quality (HQ) gene of interest plasmid to support the development of Axovia’s gene therapies for ciliopathies, including Bardet-Biedl Syndrome...
Tempe, Arizona – Cell death is fundamental to life and, thus, healthy aging. In the realm of cellular biology, ferroptosis (a form of programmed cell death) has emerged not only as a focal point of research for its potential in eliminating cancer cells, but also its role in a plethora...
Survival rates increased about 20% for children with high-risk neuroblastoma enrolled in a St. Jude Children’s Research Hospital clinical trial that included a novel monoclonal antibody produced onsite. The findings appeared today in the Journal of Clinical Oncology. Neuroblastoma is a cancer of immature nerve cells in the sympathetic nervous system. The tumor...
TEL AVIV, Israel — Chemomab Therapeutics Ltd., a clinical stage biotechnology company focused on the discovery and development of innovative therapeutics for fibro-inflammatory diseases with high unmet need, today announced early completion of patient enrollment in its Phase 2 clinical trial assessing CM-101 as a treatment for primary sclerosing cholangitis (PSC)....
BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases. “There continues to...
BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, announced today that the U.S. Food and Drug Administration (FDA) approved FILSUVEZ® (birch triterpenes) topical gel for the treatment of partial thickness wounds...
Denver, Colorado – Four children who acquired HIV in utero have remained free of detectable HIV for more than one year, according to new findings that were presented on March 6, at the Conference on Retroviruses and Opportunistic Infections (CROI) in Denver, Colo. The study is part of ongoing, multinational...
Philadelphia, PA – In a landmark study, an international consortium led by researchers at Children’s Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent...