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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…
Read moreA new large-scale genetic screening technique for spinal muscular atrophy (SMA) called comprehensive SMA analysis, or CASMA, was more effective at detecting disease-related mutations in carriers than standard approaches, a study reports. CASMA “is a simple and…
An experimental high-dose regimen of spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) is showing motor benefits for previously untreated patients of all ages, as well as those who switched over from Spinraza’s approved dose, according…
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…
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