The primary endpoint of the change from baseline in the 32-item Motor Function Measure (MFM32) total score at 12 months differed significantly between the 115 evaluable patients who were randomly assigned to receive oral risdiplam – at a daily dose of 5 mg for individuals weighing 20 kg or more and 0.25 mg/kg for those with a lower weight – and their 59 counterparts who instead received placebo.
Participants were aged 2–25 years, had confirmed 5q autosomal recessive type 2 or type 3 SMA, were nonambulant but could sit independently, and had a score of at least 2 in entry item A of the Revised Upper Limb Module.
Valeria Sansone, from the University of Milan in Italy, continues: “Clinicians should discuss risdiplam with patients and families as a new treatment opportunity with a good tolerability and safety profile, but emphasise that unanswered questions exist for use in adults with spinal muscular atrophy when considering efficacy and long-term expectations.”Įugenio Mercuri (Catholic University and Policlinico Agostino Gemelli, Rome, Italy) and co-investigators report on part 2 of the phase 2/3, double-blind SUNFISH trial, the pivotal study that led to the approval of the SMN2 pre‐mRNA splicing modifier in the USA and Europe. “The data in adults, although encouraging, need to be confirmed in larger cohorts and over longer periods than in this study.”
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