BrightInsight has launched a web-based dosing calculator to help physicians select the correct loading and maintenance dose of the treatment Hemlibra according to the body measurements of their patients with hemophilia A. The dosing calculator, launched with the support of Hemlibra’s manufacturer, Roche, will support the…

Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…

A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…

A gene therapy-based factor VIIa (FVIIa) given as prophylactic, or preventative, treatment was seen to produce specific factor levels needed to reduce the frequency of spontaneous bleeding episodes in a rat model of hemophilia A. According to scientists, this work may be a first step in developing therapies that…

A combination of Cyklokapron and Feiba — usually avoided due to a perceived risk of blood clots — appears to be safe and effective for treating patients with acquired hemophilia A (AHA), though these early study results need to be confirmed in clinical trials, researchers in Italy say. The study, “…

Recognizing risk factors of thrombosis and carefully monitoring medication doses are crucial for minimizing thrombotic complications in older patients with acquired hemophilia and serious illnesses like cardiovascular disease, a report describing four cases says. The study, “Acquired Haemophilia A. Which is the best therapeutic choice in older adults? Single…

Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…

Two new mutations affecting the production of functional clotting factor VIII were identified in a group of Vietnamese patients with hemophilia A, researchers report. Their findings were described in a letter to the editor, “Mutation characteristic of 103 haemophilia A patients in Vietnam: Identification of novel mutations,”…

The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…

A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…