An official decision on whether to approve apitegromab for spinal muscular atrophy (SMA) in the European Union may hinge on upcoming inspection results from U.S. regulators, biopharmaceutical company Scholar Rock has announced. The…
The post Rarely Speaking: SMA Beyond the Clinic appeared first on SMA News Today.
I wiped away tears of happiness and hugged my friend Andrea. She was engaged to be married and had just asked me to be her maid of honor. Of course, I said yes with all…
You know you’re in trouble when your orthopedist uses words like “sclerosis.” After several excruciating months of pain, I was exactly where I needed to be: Gillette Lifetime Specialty Healthcare in St. Paul,…
England is rolling out newborn screening for spinal muscular atrophy (SMA), which will allow all babies born in the country to be tested for the rare genetic disease shortly after birth. According to a government…
“Alyssa, does this look weird?” my mom said as she popped her head and chest out of the bathroom door. We were getting ready to go to a parade in 100 F heat and humidity.
The spinal muscles of spinal muscular atrophy (SMA) patients who also have scoliosis show abnormalities in molecular activity, particularly in biological processes that cells use to metabolize sugar molecules and protein building blocks, according to a…
My husband, Randy, and I moved to the North Carolina mountains in the closing hours of 1995. Although our children, Matthew and Katie, who at the time were 10 and 7, respectively, were initially unenthusiastic about…
A woman with spinal muscular atrophy (SMA) who has become a champion for those living with disabilities — as an author, a podcast host, and a vice president of the Muscular Dystrophy Association (MDA) —…
July is my favorite time of year to be active, travel, and chase adventures. Like many people with SMA, cold weather is hard on my body — my muscles get tense, my fingers and toes…
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