London, UK – Subtle changes in the brain, detectable through advanced imaging, blood and spinal fluid analysis, happen approximately twenty years before a clinical motor diagnosis in people with Huntington’s disease, finds a new study led by UCL researchers. The research, published in Nature Medicine, was in collaboration with experts...
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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...
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...
Charity cyclist Mark Lownes is riding almost 150 miles to raise awareness of a rare disease in memory of his “lovely” sister. The Ackworth man will lead a team of eight people on the coast-to-coast route from Cumbria to Sunderland in a bid to boost the coffers of charities which...
Smoky Mountain Harley-Davidson, in conjunction with the Dixie Iron Riders, is hosting the Cruise for 22q Charity Ride on Aug. 1 to benefit the 22q13 Deletion Foundation, which supports families affected by the Phelan-McDermid syndrome. Phelan-McDermid syndrome, also known as 22q13 Deletion syndrome, is a rare genetic anomaly caused by...
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...
LONDON, UK – CHARM Therapeutics (“CHARM”, “The Company”), today announced the advancement of CHM-029, a next-generation pan-menin inhibitor designed to overcome resistance mutations, as a development candidate for the treatment of acute myeloid leukemia (AML). It is expected that CHM-029 will enter first-in-human trials in Q2 2026. Menin inhibitors have emerged...
A little boy from Cheam has been diagnosed with a condition so rare that, since its discovery in 1965, fewer than 25 cases have been recorded worldwide. Alfie Mumford, four, suffers from the complex Gillespie syndrome, characterised by partial aniridia, meaning that part of his iris is missing and his...
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...
MELBOURNE, Australia, and MENLO PARK, California U.S.A. – ChemGenex Pharmaceuticals Limited (ChemGenex) (ASX:CXS) announced that the latest data from its pivotal study of omacetaxine in patients with T315I-positive chronic myeloid leukemia (CML) was the subject of an oral presentation and discussion today at the 2009 American Society of Clinical Oncology...