Nusinersen Wearing-Off in Adult 5q-Spinal Muscular Atrophy Patients.

ABSTRACT: The antisense oligonucleotide nusinersen was the first drug treatment available for all types of 5q-spinal muscular atrophy (SMA). The dosing regime has been derived from pivotal clinical trials in infants and children. The efficacy of nusinersen in severely affected adult SMA patients is still questionable, as no placebo-controlled trials have been conducted. In the present study, we systematically examined wearing-off phenomena during nusinersen maintenance dosing using a patient-centered approach. We found that adult SMA patients perceived wearing-off after nearly half of 51 investigated nusinersen administrations, primarily within the last month prior to the next administration. Symptoms and functions affected were mainly general strength and arm and leg muscle function next to enduran

SUBMITTER: Osmanovic A 

PROVIDER: S-EPMC7998943 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature