Hemophilia News Today

June 21, 2021June 21, 2021

Poorer Joint Health, Greater Pain Evident After COVID Lockdown

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 ... Read more

June 18, 2021June 18, 2021

Study Examines Inhibitor Risk in Non-severe Hemophilia A

Hemophilia News Today

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, ... Read more

June 16, 2021June 16, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

COVID-19 News, Hemophilia News Today

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 ... Read more

June 16, 2021June 16, 2021

Medicaid Spending on Hemophilia Therapies in US Tripled in 2005-19

Hemophilia News Today

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 ... Read more

June 14, 2021June 14, 2021

Preventive Hemlibra May Lower Hemophilia A Treatment Costs

Hemophilia News Today

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 ... Read more

June 11, 2021June 11, 2021

Man With Hemophilia A Treated for COVID-19 While on Hemlibra

COVID-19 News, Hemophilia News Today

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 ... Read more

June 9, 2021June 9, 2021

‘Awesome’ Book Contest Invites Hem B Patients to Share Their Story

Hemophilia News Today

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 ... Read more

June 7, 2021June 8, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Hemophilia News Today

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 7, 2021June 7, 2021

Partnership to Aid Access to Man-made FVIII for Patients in China

Hemophilia News Today

GenScript ProBio and Neoletix Biotechnology have entered into an agreement for clinical and commercial production of a lab-made version of coagulation factor VIII, a product that the companies hope will ... Read more

June 4, 2021June 4, 2021

Investigational Gene Therapy Increased FIX Production

Hemophilia News Today

Optimization of investigational hemophilia B gene therapy led to more than 25 times the production of factor IX (FIX), the blood-clotting protein that is missing in people with the condition, ... Read more

June 2, 2021June 2, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Hemophilia News Today

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 2, 2021June 2, 2021

MOTIVATE, Study of Treating Hemophilia A With Inhibitors, Enrolls 1st US Patient

Hemophilia News Today

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of ... Read more

May 28, 2021May 28, 2021

Gene Therapy Roctavian Prevents Bleeds Over 5 Years, Data Show

Hemophilia News Today

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data ... Read more

May 26, 2021May 26, 2021

BioMarin to Resubmit Gene Therapy Roctavian for EU Approval in June

Hemophilia News Today

The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, ... Read more

May 24, 2021May 24, 2021

Survey Outlines Top Concerns Among Hemophilia Patients in Central Europe

COVID-19 News, Hemophilia News Today

Mobility problems, bleeding, pain, and uncertainly in daily life are the biggest challenges people with hemophilia are facing, according to a survey from six central European countries. Additionally, while most ... Read more