Idelvion (albutrepenonacog alfa), a hemophilia B treatment approved for up to 14-day dosing, is now available in a large, 3500 IU vial size (3500 IU),…

While searching for content ideas for a client, I stumbled on an Instagram post that immediately caught my attention. “Human beings are seasonal,” the post stated — more or less, as I can’t recall the exact wording. But I’ve managed to internalize three key points on the subject: Life has…

Sigilon Therapeutics’s Afibromer SIG-003 candidate for the long-term treatment of patients with hemophilia B recently received an Advanced Therapy Medicinal Product (ATMP) designation in the European Union. The designation was granted by the Committee for Advanced Therapies of…

Greetings! I’m writing from Orlando, Florida, my temporary oasis from the wrath of Hurricane Ian. Last week, my mom and I evacuated to this inland city from Punta Gorda, on the coast in the southwest part of the state. Staying was not an option as my mom is oxygen-dependent…

Psychological distress triggers microscopic changes in regions of hemophiliac children’s brains that deal with emotions and cognition — and the changes show up before abnormal results on neuropsychiatric tests, researchers discovered. The team said larger studies are needed to confirm the links they found between brain changes, the stage of the…

I’ve been cleaning up my computer files and finding pictures everywhere except where they are supposed to be stored. One photograph caught me by surprise. My mighty warrior, Caeleb, went to a monster truck rally in 2014. He took a great picture with the driver of the monster…

A few years ago, I went to a tattoo convention. Before I could enter, I was required to sign a liability release. The form said that people with bleeding disorders (and a long list of other conditions) should not get tattoos. Needless to say, I am not always a…

Dimension Therapeutics will discontinue clinical development of DTX101, a gene therapy intended to treat moderate/severe-to-severe hemophilia B. The decision comes after a review of data from a Phase 1/2 clinical trial (NCT02618915), which assessed and the safety and dosing of DTX101. The trial was a…