Why carry out this study?

While pivotal trials have established the safety of risdiplam in patients with spinal muscular atrophy (SMA), evidence from real-world clinical settings, particularly in Japan, remains limited

This all-case postmarketing surveillance study aimed to evaluate the safety and usage patterns of risdiplam in Japanese patients with SMA over a 12-month period in real-world settings

What was learned from the study?

Risdiplam was generally well tolerated, with 2.2% of patients discontinuing treatment because of adverse events

The incidence of adverse drug reactions (ADRs) was 20.8%. The most common ADRs were gastrointestinal disorders (16.0%), predominantly diarrhoea (8.0%) and faeces soft (4.3%)

Advanced age, the presence of comorbidities and the use of concomitant medications may contribute to a higher incidence of gastrointestinal disorders. A multivariate analysis will be performed in the final assessment to provide a clearer understanding of the associated risk factors