WHO updates hemophilia therapies on its essential medicines list
With encouragement from the World Federation of Hemophilia (WFH) and its partners, the World Health Organization (WHO) updated its…
Articles by Lindsey Shapiro, PhD
Newly ID’d F8 mutation could lead to better hemophilia A treatments
A team of U.S. scientists discovered a naturally occurring F8 gene mutation that leads to increased activity of the factor…
FVIII from advanced liver organoids stops bleeding in hemophilia A mice
Scientists have developed advanced liver organoids — lab-grown “mini-organs” that mimic the function of organs in the body — that…
Hemophilia B gene therapy benefits last more than a decade: Study
The benefits of a one-time gene therapy were sustained for up to 13 years among men with severe hemophilia…
Treg-boosting therapy prevents inhibitors in hem A mouse model
A therapy designed to promote the growth of regulatory T-cells (Tregs) in the body prevented inhibitor development in a mouse…
Hympavzi now approved in UK for prevention of hemophilia bleeds
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) for the prevention of bleeding episodes…
Hemgenix hemophilia B gene therapy reimbursable in Germany
The cost of the one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) will now be covered for eligible…
Fitusiran, now Qfitlia, approved by FDA for hemophilia A, B patients
The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce…
Focus of NORD study: Rare disease community’s lived experiences
The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to…
Antibody therapy Mim8 seen effective in hemophilia A children
Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless…