Scholar Rock has successfully removed a manufacturing facility from its application to the U.S. Food and Drug Administration (FDA) seeking approval of apitegromab, an experimental muscle-strengthening treatment for spinal muscular atrophy (SMA). The move follows an announcement earlier this month that the company planned to remove…

MRI measurements of the facial nerve, which controls the muscles used for facial expression, may offer a simple way to detect damage to motor neurons — nerve cells that control movement — in adults with spinal muscular atrophy (SMA), according to a study. They may also reflect physical weakness…

Living with SMA entails so much more than physical challenges and caregiver shortages. Our lives are much fuller and richer than that. The riches are in our day-to-day experiences, as well as the wisdom and maturity that SMA bestows upon us. This often results in other qualities, such as…

“Did you tell your siblings my medication costs about $1,000 SGD [$783] per day, before subsidies?” I texted my dad a couple of months back. It had been years since his elder brother and sister had seen me. As fate would have it, I was due for phenol and steroid…

Infants diagnosed with spinal muscular atrophy (SMA) type 1 through newborn screening programs (NBS) and treated early with Spinraza (nusinersen) showed better survival, motor function, milestone achievement, and feeding and breathing independence than infants who began treatment after symptoms appeared, a real-world study in Turkey found. The nationwide…