Risdiplam May Be Safe and Effective in Spinal Muscular Atrophy Care

Risdiplam appears to be a safe and effective therapeutic option for patients with spinal muscular atrophy (SMA), leading to improved motor function with minimal presentation of severe adverse effects, according to a recently published meta-analysis in Pharmacotherapy.

The development of new therapeutic alternatives such as nusinersen has significantly improved the prognosis of patients with SMA. One of these drugs, risdiplam, has been proven to increase the expression of the SMN gene. Due to its recent development, trials have yet to meet the inclusion criteria for risdiplam. 

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“Therefore, this systematic review and meta-analysis aimed to estimate the efficacy of risdiplam on motor and respiratory function and the rate of treatment-related adverse events in participants with SMA,” the authors wrote. 

The studies included in the analysis used risdiplam alone or in combination with other drugs and used the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) to asses motor function improvement as well as the Hammersmith Infant Neurological Exam, the 32-item Motor Function Measure (MFM32), Revised Upper Limb Module (RULM), and Hammersmith Functional Motor Scale – Expanded (HFMSE).

After applying inclusion and exclusion criteria, the authors included 7 studies in the meta-analysis, which included 640 patients.

Results showed that CHOP-INTEND scores were over 40 in over 50% of the participants receiving risdiplam and a 100% of presymptomatic participants after a year of treatment; 12 months later, the rate increased to 76%. Similar results were observed applying the RULM score. 

“Risdiplam is an effective and safe treatment for all 3 types of SMA. It stabilizes or improves motor function in SMA1 as assessed by CHOP-INTEND and in SMA2/3 as assessed by MFM32, HFMSE, and RULM,” the authors concluded.

This article originally appeared on Rare Disease Advisor