Practical use of risdiplam (EVRYSDI▾) in Individuals with Spinal muscular atrophy

<h3></h3> Risdiplam has a tolerable safety profile, however its use has been associated with certain adverse events, which have been reported in clinical trials and the real world. Therefore, the SMA clinical community has expressed an interest for practical guidance on the management of known common adverse events. Neuromuscular experts identified three topics the SMA clinical community would particularly like guidance on; how to manage skin and gastrointestinal (GI) adverse events, and the impact of risdiplam on fertility. In October 2022, Roche organised a UK multi-disciplinary workshop which was led by UK clinicians with expertise in managing individuals with SMA. The objectives were to develop guidance for the clinical SMA community on the key considerations around fertility, and the management of GI and skin adverse events observed in individuals receiving or being considered for risdiplam. Following a review of clinical trials, case studies and real-world evidence, the clinical expert panel provided recommendations on the management of GI and skin adverse events, and guidance around fertility. The practical guidance developed by these specialists is intended to provide helpful recommendations for the management of adverse reactions in individuals with SMA receiving risdiplam. This work was sponsored and funded by Roche Products Ltd.