Adherence and Persistence Among Risdiplam-Treated Individuals with Spinal Muscular Atrophy: A Retrospective Claims Analysis.

ABSTRACT:

Introduction

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletions and/or mutations in the survival of motor neuron 1 (SMN1) gene. Risdiplam, the first and only oral SMN2 pre-mRNA splicing modifier, is US Food and Drug Administration-approved for the treatment of pediatric and adult patients with SMA. For patients with SMA, long-term adherence to and persistence with an SMA treatment may be important for achieving maximum clinical benefits. However, real-world evidence on patient adherence to and persistence with risdiplam is limited.

Methods

This retrospective study examined real-world adherence and persistence with risdiplam from a specialty pharmacy in patients with SMA over a 12-month period. Adherence was estimated by using proportion of days cove

SUBMITTER: Pineda ED 

PROVIDER: S-EPMC11133212 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature