Results 191 to 200 of about 12,413,959 (307)
Abstract Purpose Patello–femoral instability (PFI) is often caused by predisposing factors, with trochlea dysplasia (TD) as the most prominent. Untreated patellar instability leads to impaired function and an increased risk of patellofemoral osteoarthritis.
Christian Dippmann +4 more
wiley +1 more source
Significant PK variability of plasma-derived FIX concentrates in chinese children with Haemophilia B: A fixed single-dose study of factor IX-CTBB. [PDF]
Liu G +10 more
europepmc +1 more source
ABSTRACT Background Hemophilia A (HA) is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. Prophylactic FVIII replacement therapy is essential for preventing bleeds, but it carries a risk of inhibitor development, especially in previously untreated and minimally treated patients (PUPs and MTPs, respectively).
Sarina Levy‐Mendelovich +11 more
wiley +1 more source
Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components
The review presents a comprehensive overview of each component of lipid nanoparticles(LNPs)and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu +6 more
wiley +1 more source
Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. [PDF]
Male C +13 more
europepmc +1 more source
ABSTRACT Objectives We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom. Methods Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first‐time rVWF for the on‐demand ...
Mike Laffan +6 more
wiley +1 more source
Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors. [PDF]
Pochopien M +6 more
europepmc +1 more source
ABSTRACT Objectives We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) receiving recombinant von Willebrand factor (rVWF) in surgical settings in the United Kingdom. Methods Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first‐time rVWF for the ...
Mike Laffan +6 more
wiley +1 more source
Haemophilia A and haemophilia B: molecular insights (corrected version). [PDF]
openaire +1 more source
Chimeric Antigen Receptor T‐Cell Therapy: More Than an Anti‐Cancer Drug
ABSTRACT Initially, chimeric antigen receptor (CAR) T‐cell therapy was developed to eliminate malignant B cells in haematological B‐cell malignancies by targeting CD19 and B‐cell maturation antigen. This approach achieved notable success, resulting in (malignant) B‐cell depletion and inducing clinical remission in cancer patients.
Hannah C. M. Schenk +8 more
wiley +1 more source

