catheter salvage with antibiotic lock therapy for central venous port infection due to pseudoxanthomonas sp in a child with hemophilia b

Original title: “Catheter Salvage with Antibiotic Lock Therapy for Central Venous Port Infection due to Pseudoxanthomonas sp. in a Child with Hemophilia B.”

discovery and optimization of marstacimab a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia a and b

Original title: “Discovery and optimization of marstacimab, a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia A and B.”

occult hemophilia b and plastic surgery preventing bleeding events

Original title: “Occult Hemophilia B and Plastic Surgery: Preventing Bleeding Events.”

Gene therapies cost $2.1M to $4.25M. Insurance barriers and Medicaid gaps are reshaping access for rare disease families. A single infusion for a child with this fatal neurodegenerative disease carries a price tag higher than most people will earn in a lifetime. Hemgenix, approved in 2022 for hemophilia B, costs $3.5 million. Lyfgenia, for sickle cell disease, is $3.1 million. Casgevy, the first CRISPR-based gene therapy by Vertex, is $2.2 million. Bluebird bio developed gene therapies for beta-thalassemia (Zynteglo), sickle cell disease (Lyfgenia), and cerebral adrenoleukodystrophy (Skysona). These were not failed therapies. They worked. Patients got better. NewsGene TherapyHealthcare AccessRare Disease PolicyMarch 29, 2026 · Gene therapies offer cures for previously untreatable rare diseases, but prices reaching $4.25 million create devastating access barriers. Therapies that literally cure genetic diseases once thought incurable are now available. But for the families who need them most, these breakthroughs have become locked behind a pricing wall so high it is often impenetrable. The gap between scientific innovation and patient access has never been wider. A therapy that works exists, yet most patients cannot access it. This is not a hypothetical problem. It is the daily reality for families navigating a rare disease diagnosis.

Original title: “Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend”