NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the ... Read more
Updates
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Most Hemophilia Patients Show Positive Attitude for Gene Therapy in Study
Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a ... Read more -
Today I Choose Thankfulness
People are suffering. The country is divided. States are imposing limits on items that may be purchased, and businesses are closed. If there were ... Read more -
First Patient Dosed in Phase 3 Trial Testing Marstacimab, Pfizer’s TFPI Therapy
The first participant was dosed with the investigational therapy marstacimab in a Phase 3 clinical trial — still recruiting eligible patients across the globe ... Read more
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We Need a Standard of Care in Women’s Hemophilia Treatment
I visited a hemophilia treatment center for the first time in the 1980s, to ... -
Living One Day at a Time
Lately I’ve been feeling stuck. My life seems to have taken on the appearance ... -
Insurance Firm BCS Partners With NHF to Improve Healthcare Quality, Cost
The healthcare insurance company BCS Financial has partnered with the National Hemophilia Foundation (NHF) ... -
AMT-061 Increases FIX Activity, Reduces Bleeding in Phase 3 Trial, Data Show
AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased ... -
Maintaining Gratitude Through a Spirit of Thanks-living
Let’s face it. This year contains one struggle after another. We continue to ask ...
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Europe’s CHMP Recommends Approval of Hemlibra to Treat Hemophilia A
News
Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds ... Read more