Data on Spinal Muscular Atrophy Reported by Frederique Audic and Colleagues (Effect of nusinersen after 3 years of treatment in 57 young children with SMA in terms of SMN2 copy number or type).

A recent study conducted in Marseille, France, examined the effects of nusinersen treatment on children with spinal muscular atrophy (SMA) over a period of 36 months. SMA is a rare genetic neuromuscular disorder that causes muscle atrophy. The study found that 93% of the patients showed improvement in motor skills over the course of the treatment. Patients with three copies of the SMN2 gene were more likely to have better outcomes in terms of respiratory, nutritional, and orthopedic complications compared to those with two copies. However, children with two copies of SMN2 had worse motor, respiratory, and orthopedic outcomes after 3 years of treatment. This research has been peer-reviewed. [Extracted from the article]