Development of an International SMA Bulbar Assessment for Inter-professional Administration.

ABSTRACT:

Background

Progressive weakness can affect bulbar muscles in individuals with moderate to severe forms of spinal muscular atrophy (SMA). The paucity of standardized, valid bulbar assessments capturing clinically significant deficits in SMA impedes the ability to monitor function, facilitate intervention, or detect treatment response.

Objective

To fill this void, an international multidisciplinary team gathered to develop an agreed upon consensus-derived assessment of bulbar function in SMA for inter-professional administration to enhance our ability to monitor disease progression, support clinical management, and evaluate treatment effects.

Methods

Fifty-six international clinicians experienced in SMA were invited and engaged using the Delphi method over multiple roun

SUBMITTER: Dunaway Young S 

PROVIDER: S-EPMC10357133 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature