Today is a good day for me to show appreciation to my caregivers
How can I possibly show the deep appreciation that I have for the caregivers in my life with spinal muscular atrophy (SMA)? This is a question that burdens me often when I feel…
Columns
Ahead of this year’s MDA Conference, association’s CEO speaks of ‘hope’
Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the latest advances in neuromuscular disease research and…
Spinraza treatment found to restore vital amino acid in SMA type 1 children
In children with spinal muscular atrophy (SMA) type 1, the most severe form of the genetic disease, treatment with Spinraza (nusinersen) restored levels of taurine — an amino acid that plays a…
This Rare Disease Day, honor a caregiver in your life
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…
Trial of next-generation SMA gene therapy GB221 doses first patient
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…
I’m working to resolve some home equipment malfunctions
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…
The major life changes that led to our family’s SMA assignment
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…
I give back — with a little help from my friends
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…
I have a newfound appreciation for my clinic at Gillette Children’s
On a perfectly mundane February afternoon, my mom and I drove to downtown St. Paul, Minnesota. It’s a new ritual now that I’ve transitioned most of my care to Gillette Children’s Specialty Healthcare. Every six…
SMA tied to organ-specific redox imbalances in mouse study
Spinal muscular atrophy (SMA) is associated with multi-organ changes in a protective protein modification that helps guard cells against molecular damage, according to a study in mice. The researchers also found that treatment with antisense oligonucleotides —…
Recent Posts
- Mental health symptoms common in SMA patients, their caregivers
- Using my time wisely helps me balance medical care, work, and fun
- MDA Engage: Keynote speaker Mindy Henderson redefines the impossible
- Potential treatment in the works for 3 siblings with ultra rare form of SMA
- SMA treatment was once a pipe dream. Now, we have 5 options.