Fast Five Quiz: Are You Prepared to Manage Spinal Muscular Atrophy?

Stephen L. Nelson, Jr, MD, PhD


May 16, 2019

Because there is no cure for SMA, the primary goal of treatment is to slow disease progression and maximize patient quality of life and independence. SMA management is supportive and requires a multimodal and multidisciplinary approach—one that addresses patients' musculoskeletal, gastrointestinal, and respiratory complications. Supportive treatment should be aimed at improving patients' quality of life and minimizing disability.

Gabapentin, olesoxime, and riluzole were thought to have neuroprotective properties; however, no clinical trials showed any significant clinical benefits in treating patients with SMA. Likewise, although laboratory studies have shown that albuterol, hydroxyurea, phenylbutyrate, and valproic acid increase SMN transcription, clinical trials demonstrated no significant improvement in SMA disease progression.

In 2016, the US Food and Drug Administration approved nusinersen, the first drug for treating pediatric and adult patients with SMA. Nusinersen is an antisense oligonucleotide that increases production of full-length SMN protein via SMN2. Clinical trials have shown that nusinersen improves motor function and reduces mortality in patients with SMA. Although nusinersen is approved for all SMA types, clinical trials assessing its effectiveness in treating patients with SMA type IV are lacking.

It is important to note that nusinersen is a noncurative treatment; therefore, as previously noted, SMA management requires a multidisciplinary approach aimed at improving quality of life and minimizing disability, particularly in patients with slow progression.

For more on SMA treatment, read here.


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