News

Scholar Rock’s apitegromab led to motor function improvements over a year of treatment in young adults and children with spinal muscular atrophy (SMA) type 2 or 3 who could sit independently…

Treatment for 10 months with Spinraza (nusinersen) normalized the activity of 38 genes in adults with spinal muscular atrophy (SMA), a genetic study showed. Among the normalized genes were those involved in immune signaling pathways —…

Nearly two-thirds of adults in South Korea with spinal muscle atrophy (SMA) type 2 or type 3 were initially misdiagnosed, according to a recent study. In type 2 cases, the primary misdiagnosis…

Babies born prematurely were diagnosed with spinal muscular atrophy (SMA) through newborn screening programs (NBS) in Germany and, in most cases, started on treatment before disease symptoms emerged, a retrospective study reports. Doctors, however, waited to initiate…

Brain abnormalities visible on MRI scans were seven times more likely in people with spinal muscular atrophy (SMA) than in those without the disease, a small study found. Patients with indicators of more severe…

Untreated patients with spinal muscular atrophy (SMA) type 2 showed more changes in their ability to perform upper limb activities over a year than those with SMA type 3, an analysis suggests. Whether patients gained…

Michael, our patient who has spinal muscular atrophy, recently stayed at a local rehab center while his caregivers were out of town instead of having someone come stay with him like he has previously. His reasoning for this was he would have care whenever he needed it instead…

The Ministry of Health, Labour, and Welfare (MHLW) in Japan has extended the approval of Evrysdi (risdiplam) for infants genetically diagnosed with spinal muscular atrophy (SMA) who are younger than 2 months and haven’t yet had symptoms. With this extension, the treatment is now available for patients of…

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), given as a sole treatment, improves motor skills and preserves lung health and feeding abilities in infants with spinal muscular atrophy (SMA) type 1, according to a real-world study. Despite such benefits to patients treated at a mean age of 7.5 months,…

Substantial differences in newborn screening (NBS) practices for spinal muscular atrophy (SMA) across the U.S. could impact referral patterns or the timing of therapeutic interventions, a study shows. While healthcare providers evaluated newborns with positive results within the first week of life, many didn’t initiate therapy until after they…