Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found. The study, “Patients’ and parents’…
After one year, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to safely and effectively increase factor IX (FIX) activity and prevent bleeds in men with moderate to severe hemophilia B, according to new data from the Phase 3 HOPE-B trial. The men participating in the study did…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
Joint pain significantly rose and overall joint health — particularly range of motion — diminished with limitations on physical activities during the COVID-19 lockdown for people with hemophilia and joint disease, a small study from Spain reported. The study “Musculoskeletal Changes in Hemophilia Patients Subsequent to COVID−19…
Intensive treatment with factor VIII, the clotting protein missing or defective in patients with hemophilia A, is associated with a greater risk of inhibitor development among patients with non-severe disease, a study has found. The findings highlight the importance of closely monitoring these patients in order to prevent or…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled from 2005 to 2019, an analysis reported. Its researchers expect this finding will help state agencies with decisions regarding treatment coverage based on their Medicaid budget, given that the program…
Starting or switching to prophylactic, or preventive Hemlibra (emicizumab) may lead to a significant reduction in treatment-associated expenses for hemophilia A patients in the U.S., a real-world study reported. These findings highlight the cost-benefits of using Hemlibra — and may help future cost analysis studies in assessing new…
A 70-year-old man with severe hemophilia A was treated for COVID-19 while receiving prophylactic, or preventive, treatment with Hemlibra (emicizumab) to lower his bleeding risk. This case underscores the importance of close cooperation…
As part of a Coalition for Hemophilia B contest, people with hemophilia B, their siblings, and caregivers are  invited to design a comic or coloring book that tells a story about how they manage this rare blood disease. There will be a $500 award for winners in each of…
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