Treatment expectations and patient-reported outcomes of nusinersen therapy in adult spinal muscular atrophy.

ABSTRACT:

Background

The antisense-oligonucleotide (ASO) nusinersen has recently been approved as the first genetically modifying therapy for 5q-associated spinal muscular atrophy (SMA) based on randomized sham-controlled trials in infants and children. The efficacy in adults with long disease history and advanced disease status is still widely unknown; the same applies to specific expectations of adult SMA patients and to what extent they are met and may impact outcome measures.

Methods

In a longitudinal monocentric study in adult patients with SMA types 2-4, the Stanford Expectations of Treatment Scale (SETS) was assessed prior to and during nusinersen treatment. Treatment outcome was evaluated using patient-reported outcomes (PROs) as well as objectively quantifiable motor outcome

SUBMITTER: Osmanovic A 

PROVIDER: S-EPMC7359174 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature