Get regular updates to your inbox.
Atomwise, a company that specializes in using artificial intelligence (AI) to develop small molecule medicines, will partner with GC Pharma to discover new ways of treating people with hemophilia. …
Adults with mild and moderate hemophilia A have a substantial number of bleeds per year as well as joint problems and surgeries, revealing an unmet medical need in these patients, according to an interim analysis of the CHESS II study. The findings were shared at…
The European Medicines Agency (EMA) has approved 21-day dosing intervals for CSL Behring‘s Idelvion (rIX‐FP) as a prophylactic treatment for adults with hemophilia B. Idelvion — which is…
Sernova will use AgeX Therapeutics’ UniverCyte gene technology to create a cell-based therapy that delivers therapeutic levels of factor VIII (FVIII) — the blood clotting protein deficient in people with…
A new combination of gene and cell-based therapy durably delivered therapeutic levels of factor VIII (FVIII) — the blood clotting protein missing or defective in people with hemophilia A — and eased bleeding in a mouse model of the disease, a study from the HemAcure Consortium shows.
A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model of hemophilia A, a study reported. The study, “Treatment of…
Greater awareness of hemophilia and its symptoms is associated with better adherence to treatment by patients, and those with more severe disease are most likely to use a treatment as prescribed, a study based on a new questionnaire shows. Responses were seen to validate this questionnaire, which addresses…
Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — were successfully grafted into hemophiliac mice, restoring blood levels of FVIII and significantly improving…
A programmable mini-pump able to provide regular doses after surgery of FVIIa to hemophilia A patients with inhibitors — those resistant to standard FVIII replacement therapy — successfully maintained expected levels of blood clotting factors, a small study reports. Attending surgical nurses also voiced satisfaction with the pump’s use, reporting a…
A lower dose of regular, preventive therapy with factor VIII concentrates was better at reducing bleeding events and improving joint function than on-demand treatment in children with hemophilia in Thailand, a study shows. The study, “Prophylactic vs episodic treatment to prevent bleeds and preserve joint function…
Get regular updates to your inbox.