We Are the Experts of Our Own Stories
At times I struggle with documenting my journey as a caregiver for a loved one with a bleeding disorder. I can’t offer medical advice ... Read more
Updates
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#ISTH2020 – Hemlibra Continues to Show Favorable Safety Profile, Early Phase 3b Trial Data Show
When given as preventive treatment to avoid spontaneous bleeds in people with hemophilia A with inhibitors, Hemlibra (emicizumab) continues to show a favorable safety ... Read more -
Finding Joy During the Worst of the Worst
The past few months have been filled with change. My family and I moved from Texas back to New Mexico, my husband and I ... Read more -
#ISTH2020 – Gene Therapy SPK-8011 Lowers Bleeding Rate in Hemophilia A for up to 3 Years, Trial Shows
A single dose of the investigational gene therapy SPK-8011 is safe and leads to durable production of factor VIII (FVIII) for up to three ... Read more -
Life’s Struggles Are Real, but So Is Personal Growth
When my husband, Jared, and I started dating, I was aware that he has hemophilia B and epilepsy. Not long after that, someone close ... Read more
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Freeline Raises $120M for Pivotal Trial of FLT180a, a Gene Therapy Candidate for Hemophilia B
Freeline has raised $120 million to support the launch of a pivotal clinical trial ... -
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare ... -
The Roles We Play as Caregivers
Two weeks ago, my family moved back to New Mexico. Today, I find myself ... -
Being Grateful for the Boring and Mundane
I am a sucker for routine. Spontaneity does not come easily to me because ... -
Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy
CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, ... -
How to Help Your Doctor Take Hemophilia Seriously
Last week, I met a new local hematologist. He is not my main hemophilia ...
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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