In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are
currently in clinical development, gene therapy holds the promise of a lasting cure with a …

Nanocarriers as drug delivery systems are promising and becoming popular, especially for
cancer treatment. In addition to improving the pharmacokinetics of poorly soluble …

Background Fitusiran, an investigational small interfering RNA therapy, reduces
antithrombin production to rebalance hemostasis in people with hemophilia A or B, with or …

Since the evolutionary discovery of RNA interference and its utilization for gene knockdown
in mammalian cell, a remarkable progress has been achieved in small interfering RNA …

Nanocarrier-mediated protein delivery is a promising strategy for fundamental research and
therapeutic applications. However, the efficacy of the current platforms for delivery into cells …

Plasma-derived medicinal products (PDMPs) play an integral role in medical treatment and
prophylaxis. Plasma products are critical for managing bleeding emergencies, autoimmune …

The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that
can be life-threatening if untreated. Conventional therapy requires frequent intravenous …

Polymeric nanomaterials, nanogels, and solid nanoparticles can be fabricated using single
or double emulsion methods. These materials hold great promise for various biomedical …

Gene therapy provides a potential phenotypic cure for hemophilia, yet the cost of this novel
treatment is high, tempering enthusiasm and raising questions regarding cost vs benefit. To …

Introduction Recent haemophilia treatment advances include new recombinant FVIII (rFVIII)
products with improved pharmacokinetic (PK) properties that aim to reduce the burden of …