News

Gains found over 4 years with Spinraza treatment in real world

A real-world study of Spinraza (nusinersen) use in Switzerland among individuals with spinal muscular atrophy (SMA) found that, while patients tended to experience motor function benefits mostly in their first year of treatment, many continued to see gains or disease stabilization for four years. Spinraza treatment had the…

Muscle composition improves for children on Spinraza: Study

Muscle-related body composition measures improved in children with spinal muscular atrophy (SMA) types 2 and 3 who were treated with Spinraza (nusinersen), and the improvements correlated with better motor function, a study found. “Indicators of muscle mass increased in children with SMA types 2 and 3 as treatment…

Ketoacidosis not linked to diabetes in woman with SMA type 2

The case of a woman with spinal muscular atrophy (SMA) type 2 who developed an abnormal accumulation of molecules called ketone bodies was described in a recent case report in the U.S. Called ketoacidosis, this occurs when fats are broken down to obtain energy, resulting in ketone bodies being…

In memoriam, Michael Morale: Aug. 8, 1965-June 7, 2025

It is with deep sadness and heavy hearts that we announce the passing of our beloved friend, coworker, and advocate, Michael Morale. Michael died in his home in Irving, Texas, on June 7 at the age of 59. I first encountered Michael through his personal YouTube channel in 2018.

Evrysdi tablets approved as SMA treatment in Europe

The European Commission (EC) has approved a tablet formulation of spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam). “The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche,…

School-age SMA children more independent on Spinraza: Study

After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed improved motor function and more independence in tasks involving their arms and hands, a study from China has found. These motor function improvements were linked to reduced symptoms of anxiety…