Hemophilia gene therapy Beqvez reduces bleeds in Phase 3 trial
Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX…
Articles by Margarida Maia, PhD
Joint problems in hemophilia A are common despite prophylaxis
Almost two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce their…
Eloctate as prophylaxis eases pain due to hem A, improves life quality
Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for…
Roctavian nearly zeroes out bleeds, prophylaxis need over 7 years
A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis,…
Novo Nordisk buys hemophilia A program from 2seventy Bio
Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers…
AI-assisted ultrasounds may better detect joint bleeding, inflammation
Artificial intelligence (AI) can comb through ultrasound scans to detect joint bleeding and inflammation occurring as a result of…
HCB-101 shows promise for diseases due to nonsense mutations
HCB-101, hC Bioscience’s lead candidate to overwrite certain mutations that lead to hemophilia A, can reach the liver…
Cyclophosphamide therapy may be better option in some AHA cases
Added to steroids, rituximab seems to be as safe and effective as cyclophosphamide therapy at controlling bleeds and wiping…
Immune tolerance induction results don’t vary among races, ethnicities
Immune tolerance induction (ITI), a type of treatment that’s designed to eliminate neutralizing antibodies, or inhibitors, against clotting factors for…
New partners to develop universal gene therapy for hemophilia
ReciBioPharm and GeneVentiv Therapeutics are teaming up to advance the development of GENV-HEM, a gene…