An unfortunate incident recently made waves on social media here in the Philippines, where I live, when a restaurant chain posted a dismissive, sarcastic message about fake disability IDs. The poster questioned whether 30% of its customers were truly disabled and mocked those with invisible disabilities. It also reduced…
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An experimental gene therapy using modified blood stem cells helped five adults with severe hemophilia A attain therapeutic levels of clotting factor VIII (FVIII) to prevent and control bleeding, according to a small first-in-human study. Over the course of the Phase 1 clinical study (NCT05265767), none of the…
I love to watch hummingbirds. They’re lightning-fast as they move from flower to flower, feasting on nectar and insects. When they come to the feeder outside my window, their chirps and hums are different from those of other birds. I’m intrigued that the hummingbirds go into a state…
I meet a friend at noon at my home in rural northern California. We hop into my car and drive two hours north to Brookings, a coastal town just across the border in Oregon. Driving around town, I search for a quiet parking lot with good phone reception. Using a…
Metagenomi is now working on preclinical studies to advance MGX-001, its experimental gene-editing therapy for hemophilia A, with the goal of bringing the treatment candidate into clinical trials in 2026. As part of a corporate update also noting 2024 progress, Metagenomi announced that, this year, it will be…
I’m a pastor in the United Methodist Church, where the one constant we share is change. In our denomination, pastors serve on an itinerant system, which means that the cabinet (the bishop plus the district superintendents) chooses which pastors will serve in different locations. As a result, I’ll…
Prophylactic, or preventive, treatment with factor VIII (FVIII) replacement therapy for four years reduced annual bleeding rates by 91% for people with severe hemophilia A, a study from Taiwan reported. After switching from on-demand treatment to prophylaxis, patients also experienced a 90.8% reduction in annual joint bleed rates and reported…
When my husband, Jared, was younger, he was “the sick child.” With severe hemophilia B and a seizure disorder, he stood out like a sore thumb — or a rare gem. In any case, he was distinct as the child who required special accommodations at school or who missed…
Combining gene therapy with minimally invasive, robot-assisted hip replacement surgery safely and effectively treated severe joint damage for a 27-year-old man in China with hemophilia A, according to a case report. The approach significantly reduced the need for replacement therapy…
As I write this column, it’s my 32nd wedding anniversary. I met my wife, Cazandra, when we were in college. At first, we didn’t get along because we’re pretty different people: She’s an introvert while I feed off other people’s energy. College friends continue to ask us if we’re…