Therapeutic decisions under uncertainty for spinal muscular atrophy: The DECISIONS-SMA study protocol.

ABSTRACT:

Background

The therapeutic landscape for spinal muscular atrophy has changed in the last few years, encompassing respiratory/motor function and life expectancy benefits. However, physicians still have the challenge of tailoring individuals' treatment to therapeutic goals, disease progression, patient/caregiver's preferences, and personal experience to achieve an optimal risk/benefit balance. This study aims to provide insight into the preferred treatment choices of pediatric neurologists managing spinal muscular atrophy in their daily practice and to recognize behavioral factors that may influence decision-making.

Methods

This is a noninterventional, cross-sectional pilot study involving 50 pediatric neurologists managing spinal muscular atrophy in Spain. We designed an onli

SUBMITTER: Saposnik G 

PROVIDER: S-EPMC8846509 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature