The European Medicines Agency (EMA) has agreed to review a request by CSL Behring to approve the potential gene therapy EtranaDez (etranacogene dezaparvovec) for people with hemophilia B. The request, in the form of a marketing authorization application or MAA, will be reviewed under the EMA’s accelerated assessment, meaning…

I recently participated in a webinar called “Mild Matters,” organized by the Hemophilia Federation of America. The purpose was to discuss challenges faced by people with mild bleeding disorders. When the discussion turned to self-advocacy, I began to think about my own history of dealing with von Willebrand disease…

My sons reacted to hemophilia treatment in entirely different ways. My oldest son, Julian, had one port-a-cath, while my youngest son, Caeleb, had eight. Julian had two surgeries, one to place the port and the other to remove it, while Caeleb required 16 surgeries for port placements and removals.

Meaningful connections with other people are of incredible value to people with chronic illnesses. My husband, Jared, and I can both attest to this, as he lives with severe hemophilia B and a seizure disorder, while I am newly diagnosed with both bipolar II disorder and the inattentive…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

Next month, April 21–23, the MacDonald family heads to San Antonio to attend the Hemophilia Federation of America’s 2022 Symposium. I cannot wait to see old friends and participate in educational sessions regarding upcoming treatments and services to the bleeding disorders community. My wife, Cazandra, and I haven’t…

Tuesday morning, March 8. Bright rays of sun had already passed through our floor-length bedroom windows, but my sleep-deprived brain was fighting to keep my eyes shut for a few more minutes. The day before I had worked myself into a mental breakdown, which had zapped my energy completely. “Wake…

The first participant has been dosed in a Phase 1/2 dose-confirmation trial that is testing FLT180a, an experimental gene therapy that Freeline is developing to treat hemophilia B. “We are very excited about this progress in our hemophilia B program as our enhanced focus on execution is starting to…

Last week, I took Caeleb, my youngest son, to the University of New Mexico Health’s urgent care center, which is on the same floor as the Ted R. Montoya Hemophilia Program and Treatment Center. He’d been experiencing stomach and head pain for five days. I figured he had a…

As a woman affected by the bleeding disorders hemophilia B and von Willebrand disease, I realize that increasing awareness about bleeding disorders in undiagnosed women and girls is essential. All women should be aware of the symptoms of a bleeding disorder and be tested if warranted. Bleeding disorders, even mild…