In this report, our large adult center of approaching 40 patients dosed with nusinersen seeks to share our experience as well as to present lab, strength and functional objective measurements collected from our adult SMA populations dosed with nusinersen since 6/2017.

Nusinersen is a novel antisense oligonucleotide that is FDA approved for the treatment of all patients with SMA. This approval was based on functional benefit found in larger-scale, longer-term studies in the pediatric population, with more limited data available in adults. Functional benefit or possible risks of dosing this medication in the adult patient with SMA is not well-defined. Lab monitoring parameters recommended based on these studies include random urine protein and platelet count. Long term safety of nusinersen in adults in unknown. Objective strength and function measurements used to track outcomes in the pediatric studies often pertain to motor milestones and infantile activities and are not applicable or validated in the adult patient.

Data to be collected from our patient cohort of approaching 40 adults receiving nusinersen from 6/2017 to approximately date of symposium. Parameters including, serially and as applicable,: 24 hour and random urine protein, serum studies, cystatin C, platelet count, RULM score, dynamometer testing, 6 minute walk test, spirometry, CMAP. Information from patient histories, demographics, and clinic experience in administrating nusinersen program also to be presented.

Work in progress, results to be presented at the symposium.

We are presenting our experience with nusinersen in adults, and sharing the unique challenges as regards management of nusinersen program, including lab data, objective strength and function measurements, and long term safety. Conclusions in progress, to be presented at the symposium.