Routine physical therapy (PT) improved motor skills by almost 4-fold when combined with nusinersen treatment compared to nusinersen treatment alone in patients with spinal muscular atrophy (SMA), according to findings published in Frontiers in Bioscience (Landmark Edition).

SMA causes the deterioration of motor neurons, resulting in muscle weakness and decreased functional movement. In 2016, the US Food and Drug Administration approved nusinersen, a premessenger RNA therapy targeting the survival of the motor neuron 2 (SMN2) gene, to treat patients with SMA. Nusinersen significantly improves motor, respiratory, and nutritional outcomes in this patient population.

Researchers conducted a retrospective observational study in Romania to assess whether the addition of routine, intensive PT enhanced nusinersen treatment. They enrolled 55 patients with type 1 (n=20), type 2 (n=26), and type 3 (n=9) SMA. They divided the patients into 2 groups according to their ability to receive daily PT, with 39 patients in the PT-nusinersen group and 16 patients in the nusinersen-only group.

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Following evaluation by licensed pediatric physical therapists between October 2018 and June 2021 at the National Teaching Center for Children’s Neuro-Psychomotor Rehabilitation in Bucharest, Romania, patients in the PT-nusinersen group received rehabilitative treatment at least 5 days a week.

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The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score was used for patients with type 1 SMA and the Hammersmith Functional Motor Scale Extended (HFMSE) score was used for those with types 2 and 3 SMA to assess treatment efficacy in both groups at 3-time points—prior to initiation of nusinersen treatment and at 6 months and 1 year after nusinersen initiation.

The PT-nusinersen group demonstrated yields of 11.23±7.44% and 23.89±15.93% after 6 and 12 months, respectively, with a strong (0.802) and significant (P <.0001) association between yields, indicating improvements in patient motor function.

In contrast, the nusinersen-only group demonstrated yields of 2.19±1.88% and 5.37±4.39% after 6 and 12 months of treatment, respectively. While treatment with nusinersen alone still resulted in significantly improved motor function outcomes between months 6 and 12 (P <.05), the researchers found no strong (0.024) or significant (P =.929) correlation between yields, unlike the group receiving PT.

The difference between the yields of 12.66 in the PT-nusinersen group and 3.18 in the nusinersen-only group revealed an almost 4-fold enhancement of motor skill improvement between the groups.

When comparing the different types of SMA, patients with type 1 SMA showed higher yields on the CHOP-INTEND, ranging from 15.38% to 55.56%, whereas patients with types 2 and 3 SMA ranged from 5.26% to 12.96% and 12.5% to 44.44%, respectively, on the HFMSE.

“Nusinersen treatment proved to be efficient in SMA patients, as all treated patients improved their motor scores,” the authors said. “In order to achieve better motor results, we can conclude that physical therapy is strongly recommended . . . as one of the important and essential tools in SMA treatment.”


Mirea A, Leanca MC, Onose G, et al. Physical therapy and nusinersen impact on spinal muscular atrophy rehabilitative outcome. Front Biosci (Landmark Ed). 2022;27(6):179. doi:10.31083/j.fbl2706179