When I was little, SMA wasn’t widely known or understood. Its rarity meant that few people had heard of it unless they were confronted with it. Families like mine carried much of the responsibility of sharing our knowledge and experience to educate others, from friends to medical professionals, about…
The first patient has been dosed in a clinical trial testing GB221, a next-generation gene therapy for spinal muscular atrophy (SMA) type 1. “I would like to express our gratitude for the young child and family who were courageous in being the first to accept our investigational gene therapy…
Discussion
One of my rules is that nurses cannot care for me when they’re sick. Although this creates challenges, I don’t want to risk exposure. Because I live with SMA, the repercussions of a virus or other cause of illness on my health would be severe. Another thing that creates…
Damage to blood vessels in the spinal cord may play a role in nerve cell loss in spinal muscular atrophy (SMA), a new study suggests. A detailed examination of spinal cord tissue from infants who died from severe SMA detected abnormalities in the cells that line spinal cord blood…
Discussion
Countless events shape the course of our lives: marriage, career changes, parenthood, the loss of loved ones, and health crises, to name just a few. The list is almost endless. After eight years of marriage, my husband, Randy, and I decided we were ready to start a family. Overnight, my…
Discussion
I love to volunteer. If I see a need that I can meet, I am eager to jump in. And if someone asks me to help with a project or service, I am quick to say yes. So much of my life with spinal muscular atrophy (SMA) requires me…
Get regular updates to your inbox.
