Hemophilia A gene therapy: current and next-generation approaches
SW Pipe, G Gonen-Yaacovi… - Expert opinion on …, 2022 - Taylor & Francis
Introduction Hemophilia comprises a group of X-linked hemorrhagic disorders that result
from a deficiency of coagulation factors. The disorder affects mainly males and leads to …
from a deficiency of coagulation factors. The disorder affects mainly males and leads to …
Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective …
Aim To determine the immunogenicity, safety, and efficacy of rurioctocog alfa pegol in
previously untreated patients (PUPs) with severe hemophilia A (HA). Methods This …
previously untreated patients (PUPs) with severe hemophilia A (HA). Methods This …
[HTML][HTML] New orphan disease therapies from the proteome of industrial plasma processing waste-a treatment for aceruloplasminemia
A Zanardi, I Nardini, S Raia, A Conti, B Ferrini… - Communications …, 2024 - nature.com
Plasma-derived therapeutic proteins are produced through an industrial fractionation
process where proteins are purified from individual intermediates, some of which remain …
process where proteins are purified from individual intermediates, some of which remain …
Structure of blood coagulation factor VIII in complex with an anti–C1 domain pathogenic antibody inhibitor
JS Gish, L Jarvis, KC Childers… - Blood, The Journal …, 2021 - ashpublications.org
Antibody inhibitor development in hemophilia A represents the most significant complication
resulting from factor VIII (fVIII) replacement therapy. Recent studies have demonstrated that …
resulting from factor VIII (fVIII) replacement therapy. Recent studies have demonstrated that …
[HTML][HTML] Effectiveness of myofascial therapy on hemophilic arthropathy: A systematic review and meta-analysis of clinical trials
Background Recurrent hemarthrosis is one of the major complications affecting joint
functions, and causing chronic pain in hemophilia patients. Objective To summarize the …
functions, and causing chronic pain in hemophilia patients. Objective To summarize the …
Recapitulating the immune system of hemophilia A patients with inhibitors using immunodeficient mice
SC Chou, CT Yen, YL Yang, SH Chen, JD Wang… - Thrombosis …, 2024 - Elsevier
Background and aim Treating hemophilia A patients who develop inhibitors remains a
clinical challenge. A mouse model of hemophilia A can be used to test the efficacy of …
clinical challenge. A mouse model of hemophilia A can be used to test the efficacy of …
[HTML][HTML] The Impact of recombinant versus plasma-derived factor VIII concentrates on inhibitor development in previously untreated patients with hemophilia A: a 2021 …
K Kohar, SA Prayogo, L Wiyono - Cureus, 2022 - ncbi.nlm.nih.gov
Hemophilia A, the most common hereditary disorder, is caused by clotting factor deficiency.
Challenges encountered in the current treatment of hemophilia A [factor VIII (FVIII) …
Challenges encountered in the current treatment of hemophilia A [factor VIII (FVIII) …
[HTML][HTML] Clinico-epidemiological and sociodemographic profile of patients with hemophilia in the Brazilian Amazon: High prevalence of hepatitis C infection and its …
EM Santos, JM Silva, AN Barbosa… - Frontiers in Public …, 2022 - frontiersin.org
Hemophilia is a recessive genetic disease caused by a mutation on the X chromosome that
has been linked to a high risk of transfusion-transmitted infections, especially sexually …
has been linked to a high risk of transfusion-transmitted infections, especially sexually …
[HTML][HTML] Cost of hemophilia A in Brazil: a microcosting study
APB da Silva Etges, NB Schneider… - Health Economics …, 2024 - ncbi.nlm.nih.gov
Background Patients with Hemophilia are continually monitored at treatment centers to
avoid and control bleeding episodes. This study estimated the direct and indirect costs per …
avoid and control bleeding episodes. This study estimated the direct and indirect costs per …
Prediction of inhibitor development in previously untreated and minimally treated children with severe and moderately-severe hemophilia A using a machine-learning …
LL Jardim, TA Schieber, MP Santana… - Journal of Thrombosis …, 2024 - Elsevier
Background Prediction of inhibitor development in patients with hemophilia A (HA) remains
a challenge. Aim To construct a predictive model for inhibitor development in HA using a …
a challenge. Aim To construct a predictive model for inhibitor development in HA using a …