The one-time gene therapy Zolgensma (onasemnogene abeparvovec) will now be routinely accessible at low or no cost to presymptomatic infants in England who are genetically predisposed to spinal muscular atrophy (SMA). Per updated recommendations from the country’s National Institute for Health and Care Excellence (NICE), this includes…
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If it’s not my mom rocking out to “The Final Countdown” by Europe, it’s my dad reminiscing about all the cartoons we used to watch. Shows like “Arthur” and “Dragon Tales” were mainstays in the Albers household. But my favorite was “Cyberchase,” an after-school program about a bunch of kids…
“OK, SMA. You win,” I thought after signing off from a virtual meeting. I didn’t know how it was possible to feel so defeated yet relieved about finding a solution for one of my recent health woes. But there I was, staring up at my ceiling and arguing with…
Older children with spinal muscular atrophy (SMA) type 2 or 3, ages 2–5, given a single injection of Zolgensma into the spinal canal, showed clinically meaningful gains in motor function after one year, according to final data from the STRONG study. Such gains also were significantly better than…
I know I talk a big game when it comes to advocacy and setting goals for myself. The great results I achieve may lead some people to think that my efforts are effortless, but of course they never are. Surprisingly, though, some things in my life with SMA…
As I tallied the sea of tiny numbers on my tax spreadsheets, I decided to search for some background music on my computer. I love my computer — a gift from my generous family in 2020 — but it doesn’t have a CD drive. Almost all of the…
A common tendency among women and girls is to feel like we’re not enough. This insecurity is threaded throughout the media we consume. Netflix’s “One Day at a Time,” a sitcom featuring three generations of Cuban American women, comes to mind, as does the 2004 high school film “Mean…
Columnist Kevin Schaefer pays tribute to the late disability rights activist Judy Heumann, whom he interviewed in 2021.
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