People with spinal muscular atrophy (SMA) type 1 may have an increased risk of impaired kidney function, a small study suggests. The study’s findings highlight the need to further evaluate non-muscular conditions associated with SMA type 1 and to identify co-adjuvant therapies to manage them. The research was published…
Transplanting a subset of neural stem cells (NSCs) to the spinal canal may be a potential therapy to delay disease progression in children with spinal muscular atrophy with respiratory distress type 1 (SMARD1), according to a preclinical study. The study, “CSF transplantation of a specific iPSC-derived…
The severity of motor problems in children at the time of diagnosis with spinal muscular atrophy (SMA) type 2 may help to predict likely disease progression, including the start of scoliosis and required ventilation, according to an Italian observational study. This is important because a better understanding SMA’s natural history — how…
Scholar Rock has announced the publication of new preclinical data supporting the potential therapeutic value of its experimental candidate therapy, SRK-015, in improving muscle function and strength in patients with spinal muscular atrophy (SMA). The study, “Specific inhibition of myostatin activation is beneficial in mouse models of…
The use of a robotic arm controlled by electrical impulses in the arm muscles may increase the independence of paralyzed spinal muscular atrophy (SMA) patients, according to a recent case report. The case report, “Functional Tasks Performed by People with Severe Muscular Atrophy Using an sEMG Controlled…
Researchers have developed a rapid, specific blood test to screen newborns for spinal muscular atrophy (SMA) and identify carriers — those who do not have the disease but can pass it on to their children. The study, “Multiplex Droplet Digital PCR Method Applicable to Newborn Screening, Carrier…
Establishing care coordinators is key to providing successful family-centered care to young spinal muscular atrophy (SMA) type 1 patients and their families, according to a recent study. The study, “Identifying Opportunities to Provide Family-centered Care for Families With Children With Type 1 Spinal Muscular Atrophy,” was published…
Standard general anesthesia for the administration of Spinraza (nusinersen) is safe and effective in children with type 2 spinal muscular atrophy (SMA), a retrospective study shows. The study, “A retrospective cohort study of children with spinal muscular atrophy type 2 receiving anesthesia for intrathecal administration of…
Biogen’s Spinraza (nusinersen) retards disease progression in pre-symptomatic spinal muscular atrophy (SMA) infants, allowing them to achieve motor milestones they would otherwise not reach, according to updated results of a Phase 2 clinical study. The interim data were presented in a late-breaking session at the Annual Congress…
The investigational treatment risdiplam (RG7916) improved motor function in patients with type 1, 2 or 3 spinal muscular atrophy (SMA), according to preliminary results from the first part of two Phase 2/3 clinical studies. Risdiplam, being developed by Roche and Genentech in collaboration with PTC Therapeutics and the …
