Researchers have developed a new spinal muscular atrophy (SMA) screening method that’s quick, inexpensive, and easy to use with standard lab equipment. The team described their method in a paper, “A rapid and easy-to-use spinal muscular atrophy screening tool based on primers with high specificity and amplification…
Should results of a Phase 3 trial of apitegromab, a muscle-directed therapy for spinal muscular atrophy, be positive when released later this year as expected — supporting the add-on therapy being approved — apitegromab could be available to U.S. patients in 2025, Scholar Rock, its developer, announced.
Standardized measures to track the severity of spinal muscular atrophy (SMA) don’t adequately capture aspects of the disease that have major impacts on patients and their families, such as fatigue and the need for medical appointments that can be challenging to schedule, a study reports. “This study provides very…
In people with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), levels of pro-inflammatory signaling molecules tend to decrease while levels of anti-inflammatory signaling molecules tend to increase in the months after starting treatment, a study has found. Findings also suggested that changes in certain inflammation-regulating molecules may…
Treatment with Spinraza (nusinersen) led to improvements in motor function for nearly all children with spinal muscular atrophy (SMA) who received the therapy at a center in China, and among children who didn’t experience improvements, motor function was generally stable with Spinraza. The findings were detailed in a…
People with spinal muscular atrophy (SMA) who are treated with Spinraza (nusinersen) are less likely to require hospitalization or emergency medical care, and hospitalization-related costs correspondingly tend to decrease after starting on the therapy, a U.S. database study reports. The study, “Evaluation of inpatient and…
Many adults with spinal muscular atrophy (SMA) have metabolic syndrome, and those who do tend to report worse life quality and fatigue, according to a study from Germany. Nine of 36 SMA adults enrolled in the study — exactly one-quarter — met the diagnostic criteria for metabolic syndrome, or…
Survival outcomes in spinal muscular atrophy (SMA) are heavily influenced by age of onset, even among patients who are classified as having the same type of SMA, according to an analysis of more than three decades’ worth of data at a center in Thailand. In fact, data suggest…
Use of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was generally well tolerated and led to improvements in motor function for most children with spinal muscular atrophy (SMA) who were treated at centers across Brazil. “This is the first Brazilian cohort to demonstrate the benefits of [gene therapy] in…
A group of European experts has published updated recommendations for using the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in people with spinal muscular atrophy (SMA) that includes new cautions with older, heavier patients. “2024 Update: European Consensus Statement on Gene Therapy for Spinal Muscular Atrophy,” was…
