I spent a school year substituting in various special education classrooms in Fort Worth, Texas, before returning to school for teacher certification. I’d already worked with young children with developmental disabilities and loved their (usually) carefree and good-natured dispositions. My first assignment as a substitute teacher at…

The U.S. Food and Drug Administration (FDA) is giving priority review to a request that the oral therapy Evrysdi (risdiplam) be approved to treat pre-symptomatic infants with spinal muscular atrophy (SMA) who are less than 2 months old. Priority review is given to therapies with the potential to substantially…

Here in North Carolina, the weather fluctuates between springtime warmth on Christmas Day to freeze-your-butt-off cold a couple days later. We sometimes get all four seasons in the span of a few weeks, but it’s the wintertime that requires the most preparation. It forces people…

Within the U.S., there is marked state-to-state variation in guidelines for whether Medicaid will cover Spinraza (nusinersen) or Zolgensma (onasemnogene abeparvovec) treatments, according to a new study. This finding raises concerns about equitable access to these lifesaving therapies for spinal muscular atrophy (SMA), according to researchers. The…

As I write this, my room glows in the midafternoon sun. Sometime last year, I invested in a suncatcher, so there are rainbows everywhere — pastel sunbursts that are there, then gone. It’s unseasonably warm in Minnesota today, so the snow is melting, dripping steadily off the roof. According to…

Targeted next generation sequencing (NGS) — a powerful genetic method that can identify mutations in several genes simultaneously — is superior to two commonly used genetic tests for the screening of spinal muscular atrophy (SMA), a study shows. These findings highlight targeted NGS as a reliable and promising approach…