Single dose of Roctavian effective over 7 years, per new trial update
Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in…
Articles by Steve Bryson, PhD
Long-term Jivi safe and effective in hemophilia A: Real-world study
Almost three years of treatment with Jivi (damoctocog alfa pegol) reduced the annual bleeding rate among…
One-time hemophilia B gene therapy Beqvez wins OK in Canada
Health Canada has approved fidanacogene elaparvovec, formerly known as SPK-9001 and now marketed under the brand name Beqvez, for…
Fiver Liters nets $500K to develop blood, bleeding disorder therapies
Venture philanthropy fund Pathway to Cures (P2C)) has invested $500,000 in Five Liters, which is developing noninvasive, nonpharmacological neuromodulation…
Early study uncovers why FVIII inhibitors may develop in Hem A
The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on…
Canada OKs Alhemo for hemophilia A patients with inhibitors
Novo Nordisk’s Alhemo (concizumab injection) has been approved in Canada for hemophilia A patients, 12 years or older,…
In nonsevere hem A, Hemlibra not as cost-effective as standard care
Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in…
FDA grants fast track status to hemophilia B treatment SerpinPC
The U.S. Food and Drug Administration (FDA) has granted fast track designation to SerpinPC, Centessa Pharmaceuticals’ investigational treatment for people…
Hemgenix seen to reduce bleeds better in hemophilia B in new data
Hemgenix (etranacogene dezaparvovec), the first gene therapy approved for adults with hemophilia B, has been shown to work…
EU approves Hemgenix, one-time gene therapy for hemophilia B
The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia…