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BETHESDA, Md. – Today, the CF Foundation announced a new collaboration with venture creator Deep Science Ventures, focused on uncovering and designing new technologies with the potential to overcome challenges to developing genetic therapies for cystic fibrosis. The collaboration is part of the Foundation’s $500 million Path to a Cure to accelerate...
Shenzhen, China – Gestational diabetes mellitus (GDM) is a prevalent pregnancy complication posing significant health risks to both mothers and their newborns. Early detection and treatment of GDM are crucial to prevent adverse outcomes. Current screening methods, like glucose tolerance tests, are in after 24 weeks of pregnancy and have...
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...
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...