Thank goodness for the great friendships that we forge along the way. Those of us who live with chronic illnesses learn the most about caring for our loved ones from those who are in our communities. While teaming with medical staff is crucial to the diagnosis and treatment…

Durveqtix (fidanacogene elaparvovec), a gene therapy for adults with severe and moderately severe hemophilia B, has been granted conditional marketing authorization by regulators in the European Union. The decision by the European Commission follows a positive recommendation on the …

Commuting in a crowded metropolis can be a daunting experience. Many cities around the world struggle with inadequate public transportation infrastructure, poor urban planning, and heavily congested roads. In places like metropolitan Manila, where we live in the Philippines, navigating public transportation can feel like a game of survival.

Last in a series. Read parts one and two. For 21-year-old Domenic Catrine, hemophilia B once dictated every part of life, from wearing a helmet during childhood playtime to college routines shaped by weekly factor infusions. Then he underwent gene therapy during his sophomore year in…

The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s Investigational New Drug (IND) application for its gene therapy candidate to treat severe hemophilia A. FDA’s decision on BMN 270, also known for its generic name valoctocogene roxaparvovec,…

I thought the worst days were behind me in helping my youngest son navigate his bleeding disorder. Caeleb is 16 years old and a junior in high school. During his younger years, an inhibitor wreaked havoc on my son, causing extreme complications and joint bleeds that took an…

Several genetic and environmental factors can play a role in the development of inhibitors against treatment with factor VIII in hemophilia A patients, according to a review study. The review, “Risk factors for inhibitor development in severe hemophilia A,” was published in the journal Thrombosis Research. Severe…

Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…

Re-administering a viral vector-based gene therapy for hemophilia A was shown to be safe and to achieve long-term improvement in a dog model of the disease — even after neutralizing antibodies were evident following the initial treatment. The study, “An observational study from long-term AAV re-administration in…

In my years of discussing my sons’ bleeding disorders here, I too have had a hereditary disorder: essential tremors, which I inherited from my mother’s side of the family. While my disorder isn’t life-threatening, several factors seem eerily similar to my sons’ struggles with hemophilia. My tremors don’t…