The Canadian Agency for Drugs and Technologies in Health (CADTH) will not recommend reimbursement for Zolgensma in children with spinal muscular atrophy (SMA) who are more than 6 months old, citing insufficient evidence of efficacy and safety in older patients. “Other countries are providing guidance and funding based…

Difficulties with biting and fatigue while chewing are common among patients with type 2 and type 3 spinal muscular atrophy (SMA) who are unable to walk unassisted, a Dutch study reports. The findings highlight the need for interventions to help maintain muscle strength in the jaw of these patients…

Zolgensma may result in faster and greater functional improvements than Spinraza (nusinersen) in infants with spinal muscular atrophy (SMA) treated up to 2 years of age, according to a survey of 22 health providers in the U.S. Given the survey’s limitations, such as its small sample and subjective…

Children with spinal muscular atrophy (SMA) type 1 may have neurodegeneration in several areas of the brain, with impaired cognitive function and profound speech difficulties, according to a review study. However, brain involvement in this severe type of SMA is understudied and further research — using objective measures and…

Despite recent treatment advances, feeding difficulties are still one of the most common complications for infants with spinal muscular atrophy (SMA) type 1, an observational study has found. The study, “Feeding and Swallowing Problems in Infants with Spinal Muscular Atrophy Type 1: an Observational…