News

Young children with severe spinal muscular atrophy (SMA) have substantial loss of motor neurons in the thoracic region of the spinal cord, the area between the neck and the bottom of the ribs, a study found.

Taldefgrobep alfa did not significantly improve motor function across the total population of treated spinal muscular atrophy (SMA) patients in a pivotal Phase 3 clinical trial, but significant improvements were seen among Caucasian participants. That’s…

A patient I work with who has spinal muscular atrophy, Michael, continues to experience pain in his right hip with prolonged sitting since moving to a new wheelchair earlier this year. He has continued to lose muscle mass in his hip, leading to increased pressure on his sciatic…

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…

At Cure SMA’s 13th annual Hope on the Hill event in Washington, D.C., spinal muscular atrophy (SMA) patients, their families, lawmakers, and other advocates gathered to celebrate recent progress in disease research and legislative…

Slightly more than half of spinal muscular atrophy (SMA) patients in Australia who gave feedback for a study on how well they thought their treatments worked did not notice any improvement. The researchers said the results suggest a…

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…

The muscle fibers of children with spinal muscular atrophy (SMA) type 1 are substantially different from those of age-matched peers early in life, a study found. Muscle fibers are the basic units of muscle tissue that…

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.