It’s that time of year again when brackets are busted, Cinderella stories happen, and basketball fans everywhere are glued to the television for the madness of March. The NCAA basketball tournament is full of buzzer-beaters, heartbreak, and jaw-dropping comebacks. And oddly enough, for many of us living with bleeding disorders,…
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Adults across the general public in Canada think an under-the-skin injection treatment for hemophilia A would be preferable to those needing to be infused intravenously, or directly into a vein, a study based on interviews reported. Results of the analysis, sponsored by Roche Canada and conducted by Broadstreet…
Years ago, I attended a bleeding disorders conference where a person with hemophilia shared about an encounter they had with a law enforcement officer during a routine traffic stop. In order to appreciate the exchange, it’s important to understand that those of us with hemophilia are missing a protein…
Women like me often wonder how the hemophilia community has overlooked us for so long. The stories of women labeled as mere “carriers” — despite experiencing life-threatening bleeding episodes — are endless. For decades, we’ve lived with symptoms that disrupt our lives, yet we were dismissed, misdiagnosed, or told…
Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe hemophilia A in the Phase 3 GENEr8-1 study were off standard replacement therapies and did not experience any bleeds requiring treatment. That’s according to new data announced in a…
I still need to select my one-word resolution for 2022. I usually have an idea by the end of November, but 2021 was busy and fruitful, and thinking about a word was not on my radar. I relished every moment of the year because I genuinely found peace. Instead…
SB-FIX, developed by Sangamo Therapeutics, is an investigative gene therapy for hemophilia B that, by making changes to the DNA sequence, may allow hemophilia B patients to make their own factor IX protein. (Factor IX is missing or present at low levels only in patients with hemophilia B,…
Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more expression, or activity, of genes involved in activating the immune system, a small study reports. “The results of our study reveal that there is an upregulation of genes involved with…
Despite what appears to be a tranquil season regarding my sons’ hemophilia, I know it simmers right under the surface and could rear its ugly head immediately. For now, life is good. As my wife, Cazandra, and I are moving a couple hundred miles away as we…
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…