Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis.

ABSTRACT:

Introduction

Spinal muscular atrophy (SMA) is a genetic, neuromuscular disease caused by deletions and/or mutations in the survival of motor neuron 1 (SMN1) gene leading to reduced SMN protein levels. Nusinersen, an intrathecally administered antisense oligonucleotide therapy that increases SMN protein levels, is approved for use in adult and pediatric patients with SMA. Data to inform real-world patient adherence and persistence to nusinersen are limited, with disparities in the population with SMA, study design, and results. The objective of this study is to characterize real-world nusinersen adherence and persistence in patients with SMA.

Methods

This retrospective study examined nusinersen adherence and persistence over a 2-year period in patients with SMA in the USA fro

SUBMITTER: Fox D 

PROVIDER: S-EPMC9988750 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature