A study evaluates the completeness of a medical record dataset linked to administrative claims for patients with hemophilia B. This research highlights the potential of tokenization in improving data accuracy and accessibility for rare disease management.

A study highlights the use of antibiotic lock therapy for salvaging central venous ports infected with Pseudoxanthomonas in a child with hemophilia B. This approach may offer insights into managing infections in pediatric patients with rare diseases.

Research highlights the discovery and optimization of marstacimab, a human monoclonal antibody designed to target tissue factor pathway inhibitor for treating hemophilia A and B. This advancement could lead to new therapeutic options for patients with these bleeding disorders.

Gene therapies are priced between $2.1M and $4.25M, creating significant access barriers for families affected by rare diseases. Notable therapies include Hemgenix for hemophilia B at $3.5M and Lyfgenia for sickle cell disease at $3.1M, raising concerns about insurance coverage and Medicaid gaps.