Sometimes it feels like I’m living two separate lives. I’m very intentional when it comes to social media. My Instagram account is a carefully curated feed of Reels, cat pictures, and pastel-colored infographics. This is my cottagecore life. I wear dresses, listen to classical music, and wish I…

Note: This column includes thoughts about suicide. Resources for help are listed at the end of the column. On a brown and white display cabinet in my living room are two framed pictures of me. One was taken in a professional studio when I was about 6 years old. I’m…

In the first year of a newborn screening program for spinal muscular atrophy (SMA) in Japan, more than 10,000 babies were screened and one tested positive for the disease, according to a new study. The baby with SMA was able to receive the gene therapy Zolgensma (onasemnogene abeparvovec-xioi)…

Compared to what would be expected without treatment, the approved oral therapy Evrysdi (risdiplam) led to marked benefits in survival and motor outcomes for babies with spinal muscular atrophy (SMA) type 1, according to a comparison of clinical trial data. “Untreated infants with Type 1 SMA … reached…

Moxifloxacin, an antibiotic with a well-established safety profile, boosted production of the SMN protein in cellular models of spinal muscular atrophy (SMA) and it extended the lifespan and improved motor function in mice with the disease, a new study reports. “Preclinical data from patient-derived MNs [motor neurons] and a…

SMA Awareness Month is upon us! To celebrate and remind everyone that raising awareness includes talking about our daily lives and relationships, I’m honored my best friend, Brianna Albers, said yes to the crazy idea of us interviewing and challenging each other publicly. Please read her interview with…

Initiating a disease-modifying therapy (DMT) before the onset of symptoms and at the earliest time possible results in the best outcomes in infants with spinal muscular atrophy (SMA), with most achieving otherwise unachievable motor milestones and within the normal age range. This is particularly crucial for infants likely…

For most of his life, Jose Flores avoided interacting with the spinal muscular atrophy (SMA) community because he felt out of touch with it. An SMA type 4 patient, Flores didn’t need a wheelchair until he was 22, and found it difficult emotionally to see children who weren’t…

Longtime readers of my column will know that I am fellow columnist Sherry Toh’s biggest fan. Navigating SMA alongside her has been one of the greatest honors of my life. This week, Sherry and I thought it would be fun to interview each other — not only…

It’s been a minute since I put my degree in counseling to good use. Let’s change that, shall we? Last week, I wrote about a recent ghost vacation. For those who didn’t read that column, here’s the gist: I came back from this year’s Cure SMA conference with a…