Do you remember the summer of 1998? It’s been a while, so let me remind you. It was the season of blockbuster movies like “Saving Private Ryan” and “The Mask of Zorro.” The airwaves were filled with hits by stars like the Backstreet Boys and Shania Twain,…
Discussion
Treatment before birth with RG7916, a compound similar to Evrysdi (risdiplam), improved weight, muscle health, motor function, and survival in a mouse model of severe spinal muscular atrophy (SMA), a study found. However, treatment given only before birth, or prenatally, did not fully preserve numbers of motor neurons…
Discussion
The Muscular Dystrophy Association (MDA) brought its Engage Community Seminar to Hershey, Pennsylvania, on July 18, uniting individuals with neuromuscular diseases, caregivers, and medical experts for a day of education and connection. Hosted in collaboration with the Penn State Health Milton S. Hershey Medical Center, the one-day event featured a…
Discussion
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…
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