Several studies presented at the ICNA/CNS 2020 virtual conference provided new data on treatments for spinal muscular atrophy (SMA).
Dr Jerry Mendell from the Center for Gene Therapy at the Nationwide Children’s Hospital in Columbus, Ohio, USA, reported on the long-term follow-up of 13 patients with SMA type 1 treated with the gene therapy onasemnogene abeparvovec.
He said a sustained, durable response was achieved for up to 5.2 years (in the therapeutic dose cohort) after dosing, in patients up to 5.6 years of age. Motor milestones and survival were maintained in the therapeutic dose cohort, and two patients gained a milestone.
In a separate presentation of interim results from the STR1VE-EU study of onasemnogene abeparvovec, researchers reported that 21 of 32 treated patients (65.6%) with SMA type 1 had already achieved developmental motor milestones not observed in the natural history of the condition. The occurrence of adverse events was consistent with previous reports.