Risdiplam for the treatment of adults with spinal muscular atrophy: Experience of the Northern Ireland neuromuscular service.
Gavin McCluskeySiobhan LambSarah MasonGrainne NicFhirleinnIsobel DouglasSandya TirupathiKaren E MorrisonJohn McConvillePublished in: Muscle & nerve (2022)
Our study raises hopes that risdiplam may be efficacious in adults; however, more systematic studies in larger cohorts are needed before drawing any definitive conclusions. This study also demonstrated the feasibility of virtual assessments.