Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...

5q-spinal muscular atrophy (5q-SMA) is one of the more common types of spinal muscular atrophy (SMA) affecting around one in ten thousand individuals worldwide. Currently, tests are unable to detect ...

Amsterdam, November 15, 2023 – 5q-spinal muscular atrophy (5q-SMA) is one of the more common types of spinal muscular atrophy (SMA) affecting around one in ten thousand individuals worldwide.

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

"Our findings suggest that patients with spinal muscular atrophy experience various types of respiratory events, which cannot be differentiated by oximetry. Thus, polysomnography combined with ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research. A ...

Please provide your email address to receive an email when new articles are posted on . An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular ...

Spinal muscular atrophy (SMA) Type 1 is a rare but serious genetic condition that weakens muscles and can make basic activities like eating and breathing hard for babies. Early treatment—especially ...

Salanersen (BIIB115/ION306) is a novel antisense oligonucleotide (ASO) with the potential to achieve high efficacy and once yearly dosing in spinal muscular atrophy (SMA) Interim Phase 1 data show ...