Spinal Muscular Atrophy, Sleep-Disordered Breathing, and the Effects of Disease-Modifying Therapies: A Narrative Review.

What was studied

This narrative review looked at sleep-disordered breathing in spinal muscular atrophy. It covered how common the problem is and what may cause it. It also looked at disease-modifying therapies. The authors searched the literature.

What they found

Sleep-disordered breathing was most common in the first year after diagnosis. Central and mixed sleep apnea made up a notable share. Disease-modifying therapies had varied to positive effects. They improved but did not remove the breathing problems. The evidence favored nusinersen most strongly. Data for risdiplam were scarce.

What it means for patients

The authors say large, blinded, multicenter studies are essential. They want to know how the therapies affect care and prevention. They also call for study of single-agent versus combination therapy.

Why it is not certain yet

Much of the evidence came from small studies and abstracts. Methods varied in rigor. The abstract gives no numbers. Data for risdiplam were scarce. A narrative review does not pool results. The data come from other studies. Findings may change as more data arrive.

Source

Spinal Muscular Atrophy, Sleep-Disordered Breathing, and the Effects of Disease-Modifying Therapies: A Narrative Review.

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