Zolgensma

It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, more than 95% of babies diagnosed with…

Children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in infancy are maintaining motor milestones after up to a decade of follow-up, according to new long-term clinical trial data. “At their…

Treatment with the approved gene therapy Zolgensma was found to ease breathing difficulties in children with spinal muscle atrophy (SMA), reducing hospitalizations due to respiratory issues by than 25%, according to a real-world study in Israel.

Some 70% of children with spinal muscle atrophy (SMA) type 1 who underwent treatment with Spinraza alone or with Spinraza and then Zolgensma reached some motor milestones over 18 months, with greater improvements in those who received Spinraza…

Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, according to a recent study from Europe.

Babies born prematurely were diagnosed with spinal muscular atrophy (SMA) through newborn screening programs (NBS) in Germany and, in most cases, started on treatment before disease symptoms emerged, a retrospective study reports. Doctors, however, waited to initiate…

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), given as a sole treatment, improves motor skills and preserves lung health and feeding abilities in infants with spinal muscular atrophy (SMA) type 1, according to a real-world study. Despite such benefits to patients treated at a mean age of 7.5 months,…

Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improves motor function in children with spinal muscular atrophy (SMA) who carry at least four copies of the so-called backup SMN2 gene, according to real-world data from the RESTORE patient registry. SMA children treated with the approved gene therapy achieved several motor…

Following treatment with gene therapy Zolgensma (onasemnogene abeparvovec-xioi), adding a second therapy, such as Spinraza (nusinersen) or Evrysdi (risdiplam), did not prevent widespread muscle disease progression in infants at risk of spinal muscular atrophy (SMA) type 1, a real-world study found. Although children treated with…

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]…