Author Archives: Katherine Poinsatte

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.

Gene therapy that selectively delivers the SMN1 gene to the liver reduced alterations related to spinal muscular atrophy (SMA) in a mouse model, restoring muscle size and correcting pancreatic issues. Mice that received liver-specific SMN1 also lived longer, performed…

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…

The use of advanced genetic testing techniques helped clinicians in China to identify novel mutations in the SMN1 gene in two unrelated children — and thus correctly diagnose them…

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.