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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 McConville
Published 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.
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