New swimming trunks, sunscreen, cap, sleeping bag — gathering these items means it’s time for hemophilia camp! My son Caeleb is so excited that he is driving his dad and me a little crazy. Two years ago, he broke his wrist the night before camp and wasn’t…
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Sangamo Gets OK to Enroll UK Patients in Phase 1/2 Trial on SB-FIX, Potential Hemophilia B Therapy
A Phase 1/2 clinical trial on SB-FIX, an investigative gene-editing therapy for hemophilia B, is now authorized to enroll adult and adolescent patients in the U.K., Sangamo Therapeutics announced. Approval from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) will make this the first genome-editing study conducted in Europe. Once safety…
I’m grieving for my youngest son, Caeleb, a high school senior. Caeleb’s academic pursuits haven’t been successful, according to the world. Society revels in straight A’s, being in the top 10% of a graduating class, and receiving full-ride scholarships to college. If a student isn’t meeting these expectations and doesn’t…
A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…
Freeline has raised $120 million to support the launch of a pivotal clinical trial of FLT180a, the company’s lead gene therapy candidate for the treatment of hemophilia B. FLT180a uses AAVS3, Freeline’s proprietary adeno-associated virus (AAV) protein shell, or capsid, to infect human liver…
My husband, Jared, lives with hemophilia B and a seizure disorder. He also lifts weights, performs bodyweight workouts, and swims. Now and then, someone will see him doing laps in the pool and ask, “Are you a professional?” To which he responds, “No, the water just loves me more.
During my husband Jared’s recent hospitalization, I heard one particular sentence more times than I can count: “We’ll wait for Doc’s approval.” Jared was admitted for a burn on his hand, an injury that, on its own, often follows a fairly standard treatment path. But because he lives with…
New research suggests that the main developmental role of heterochromatin, a form of dense DNA found in chromosomes, is to suppress virus-like DNA elements known as “jumping genes” from replicating and attaching themselves across a person’s genome, potentially destroying important genes and causing a variety of diseases, including hemophilia and cancer. The study, by University of North Carolina…
Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…