A woman with spinal muscular atrophy (SMA) type 3 experienced a mild decline in motor function after pausing Spinraza (nusinersen) during her pregnancy. After she restarted treatment following delivery, however, her motor function stabilized and her walking endurance partially recovered. Overall, the case suggests that women with milder SMA and better-preserved…
Discussion
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 European Medicines Agency (EMA) is currently reviewing the experimental muscle-strengthening therapy. However, European authorities are…
Discussion
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 the joy in my heart! I love both her and her fiancé, watched them grow…
Discussion
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, Minnesota. I’d been dealing with hip and lower back pain since early…
Discussion
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 press release, the program is already ahead of schedule, and some labs will…
Discussion
“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. Trying to dress in as few layers as possible was definitely a requirement. However, my…
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