Showing 2917 results for "hemophilia"

Stepping Out of the Echo Chamber Is Crucial for Advocacy

It’s election season here in the Philippines, and two presidential candidates, both with large bases of support, are going head-to-head. In light of this, I came across a social media post reminding people to get out of their “echo chambers” and make their voices heard during this election cycle. The…

Enrollment Complete for GENEr8-3 Trial of Roctavian Plus Steroids

BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…

Sometimes the Best Answer Is ‘No’

I knew that I wanted to teach theology at the university level. I prepared a great résumé and felt academically equipped, having received my doctorate in ministry. I applied to dozens of schools, but no one offered to hire me. I questioned my abilities and couldn’t understand why I…

Teachable Moments May Change Perspectives

I went to college to become a teacher. Actually, to become a middle school band director. Four years of college were great, but student teaching was how I learned to teach. Two excellent supervisory teachers helped me find my way, answered questions, and taught me useful lessons by their example.

Gene Therapy EtranaDez Allows Hem B Patients to Stop Prophylaxis

Nearly all men with moderate-to-severe hemophilia B given the experimental gene therapy EtranaDez (etranacogene dezaparvovec) in the HOPE-B trial have stopped using prophylactic therapies, full study results show. Trial data also indicated that EtranaDez, formerly known as AMT-061, is effective in people with antibodies against the viral vector…

Reflections on Pain After an Odd Jaw Bleed

It happened after a month of celebrations. First, it was Christmas and New Year’s. Then, it was my husband Jared’s birthday. Five days later, our daughter, Cittie, turned 3. Plus, we had just moved to a new home next to some uncles who kept inviting us to relatives’ birthdays…