A community-driven campaign called SMAshing My Limits And Going Places aims to make it easier for people with spinal muscular atrophy (SMA) to find accessible places to stay when they travel. The campaign is spearheaded by Becoming…
Last month, I wrote about a whiteboard of collected memories I received as a gift when I graduated from college in 2005. It had 38 pictures on it that showcased the many life adventures…
Ready or not, we’re about to say “Adios!” to another month. Our daughter, Katie, and son-in-law, Paul, arrive today for a whirlwind visit, sending July packing with a bang. A few hours after Katie and Paul…
I had a date with Count Dracula. An award-winning ballet adaptation of Bram Stoker’s immortal creation — “Dracula: Ballet at its Darkest” — was to arrive in my homeland of Singapore for…
A combined newborn screening test for two rare diseases — spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID), a condition that leaves infants with little or no working immune systems — correctly identified every affected baby, with…
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…
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…
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…
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…
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…
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