The smell of rubber. The clanging of metal plates against one another. The sight of sweaty bodies heaving, struggling. Towels slung over shoulders. Muscles popping out of the chiseled physiques of a couple who walk through the place like a king and queen (fittingly, as they are its owners). Jared…

Yesterday, I went through the pictures on my iPhone. I started with the most recent, moving backward, my heart beating faster with each swipe. By the time I had viewed two years’ worth, anxiety had me in its grasp. I felt like I couldn’t breathe, and I stopped looking…

The start of a new school year is one of my favorite times. It smells of new pencils and crayons, lunchboxes, and my favorite, spiral notebooks. It’s a season of new beginnings, and when you have a child with hemophilia, you need the adults who will be teaching them…

uniQure has achieved its target patient enrollment goal ahead of schedule for its ongoing Phase 3 HOPE-B trial. The trial is assessing the safety and efficacy of AMT-061 (etranacogene dezaparvovec), the company’s investigational gene therapy for the treatment of patients with moderate and severe…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

There is no doubt that the new medicine Hemlibra (emicizumab) is a game changer in the treatment of hemophilia. Instead of multiple infusions daily, one injection could last two weeks to a month. Such incredible new ways of approaching treatment and care introduce different concerns and result in…

After over a year without a gym membership, my wife, Cza, and I are back in our lifting den. We do it for fun and, of course, fitness. I’ve had several injuries during the past few months, all of which I can associate with the lack of physical exercise…

Caregiving is hard work. When your child is chronically ill, caregiving goes beyond the expected parental duties. You have overwhelming responsibilities. There are doctor’s appointments, infusions, and treatments at home. There are hospital stays, lab work, and missed school days. Keeping up with your child’s medical condition can…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

For many years we tried to overcome my younger son’s reaction to factor VIII. No matter which medical product we tried, we ended up with the same results. He would break out in hives early in the infusion. We learned that my son had developed an allergy to factor VIII,…