Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients

NOVEMBER 13, 2020 

Spinraza (nusinersen) effectively preserves respiratory muscle strength in addition to motor function in children with spinal muscular atrophy (SMA) type 2, according to a real-life, small study in France.

Larger studies are needed to confirm these findings, and to determine the usefulness of age-adapted respiratory muscle assessment as an efficacy measure in SMA clinical trials, the researchers noted.

The study, “Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen,” was published in the journal Pediatric Pulmonology.

Developed by BiogenSpinraza was the first disease-modifying therapy approved for all types of SMA. It is administered directly into the spinal canal at a recommended regimen of four doses (12 mg each) in the first two months, followed by maintenance treatment at the same dose every four months.

Several clinical and real-life studies have shown that Spinraza safely and effectively improves motor function and survival among children with SMA. However, its effects on respiratory muscle strength — which is progressively lost in these patients, leading to respiratory failure — remain largely unexplored.

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