Self-care Isn’t Selfish
When you become a parent, your world instantly changes. Your life focus becomes the child that you have brought into the world. When your ... Read more
Updates
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Spark Therapeutics Provides Update on SPK-8011, Other Potential Gene Therapies for Hemophilia
SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual ... Read more -
At Times, Letting Go of Perfection Is the Perfect Decision
All of the items I own are covered with breast milk; it’s on my clothes, bedsheets, even my phone. Its subtle sweetness occasionally attracts ... Read more -
Hemlibra Now Available to Patients in Developing Countries via Aid Program
Hemlibra (emicizumab), the sole approved prophylactic treatment for hemophilia A, is now available to patients in developing countries via the World Federation of Hemophilia (WFH) Humanitarian ... Read more -
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
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Preliminary Data Further Show MarzAA’s Potential to Prevent Bleedings
Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) ... -
We Will Continue to Rebuild Our Lives
February 2014 proved to host the last bleed “MacDonald the Younger” faced thus far. ... -
Single Dose of Gene Therapy AMT-061 Proves Effective in Hemophilia B
A single administration of AMT-061, an investigational gene therapy for people with severe and ... -
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What Are the Things That Most Defines Us?
The following is a group activity I like to do in some of the ... -
To My Husband, the Father of My Daughter
To the father of my daughter, Thank you for being present from the very ...
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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 ... Read more