A Canadian Adult Spinal Muscular Atrophy Outcome Measures Toolkit: Results of a National Consensus using a Modified Delphi Method.

ABSTRACT:

Background

Spinal Muscular Atrophy (SMA) is a rare disease that affects 1 in 11 000 live births. Recent developments in SMA treatments have included new disease-modifying therapies that require high quality data to inform decisions around initiation and continuation of therapy. In Canada, there are no nationally agreed upon outcome measures (OM) used in adult SMA. Standardization of OM is essential to obtain high quality data that is comparable among neuromuscular clinics.

Objective

To develop a recommended toolkit and timing of OM for assessment of adults with SMA.

Methods

A modified delphi method consisting of 2 virtual voting rounds followed by a virtual conference was utilized with a panel of expert clinicians treating adult SMA across Canada.

Results

A con

SUBMITTER: Slayter J 

PROVIDER: S-EPMC8385500 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature