A preventive treatment for hemophilia B called dalcinonacog alfa (DalcA) led to an increase of more than 12% in the levels of factor IX in two patients with severe disease treated in a Phase 2 trial, reaching the study’s main goal. Researchers also found no immune reaction against the therapy. Enrollment is…

Years ago, I watched my youngest son, “MacDonald the Younger,” as he struggled with recurring joint bleeds. Each episode took more of his mobility away. He could not extend his leg beyond 45 degrees because his knee was so full of blood, and he used a wheelchair for at least…

Last year was difficult for my mighty warrior Caeleb. Moving to a new school in a town where everyone knows everyone is not something you wish for as an incoming seventh-grader. This year was better until I discovered that a couple of boys were bullying Caeleb and he did not…

The gene therapies AMT-060 and AMT-061 both restored coagulation factor IX levels in a primate model of hemophilia B, but AMT-061 resulted in greater coagulation activity at the same dose, a new study reports. The study, “Enhanced Factor IX Activity Following Administration of AAV5-R338L “Padua” Factor IX…

Recently, I’ve been reading a lot of online content about hemophilia on forums dedicated to the disease and in articles. It could be because my Google Alerts and scholar searches are sending me plentiful links to browse. I’m noticing some consistent themes online: Moms are desperate for solutions;…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Health insurance is largely effective for Chinese patients with rare diseases such as hemophilia when it comes to being able to utilize healthcare services and buy medication. However, affordable and accessible treatment is still lacking and there is still a need for improvement in diagnostics and treatment of these rare…

When “MacDonald the Younger” was 5, a social worker from the hemophilia treatment center gave his name to the Make-A-Wish Foundation. They granted my son’s wish to go to Disney World. I couldn’t believe that a 5-year-old child would want to go to a theme park filled with great…

I’m confined to my bed, suffering from a bleed once more. Many hemophiliacs dread having a bleed in this area, called the iliopsoas muscles. It’s considered a very serious bleed, as it can result in permanent femoral nerve damage and, in some cases, amputation of the leg. The…

My 13-year-old son, Caeleb, is a victim of bullying. This school year had a better start than the previous one until a couple of boys decided to pick on my mighty warrior. Name-calling and lies are hurtful, but at least words don’t leave marks on our bodies. But when…