Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada.

ABSTRACT:

Background

Spinal muscular atrophy (SMA) is a rare neurodegenerative disease characterized by progressive muscular weakness, which occurs in one in 6,000 to 10,000 live births. The burden of SMA on Canadian patients and caregivers is not known.

Objective

To characterize the burden of SMA in Canada as reported by patients and caregivers, including disease and treatment impacts, indirect costs, and caregiver burden.

Methods

Surveys were distributed by Cure SMA Canada and Muscular Dystrophy Canada to individuals with SMA and their caregivers. The online surveys were anonymous and completed between January 28 and February 21, 2020.

Results

965 patient and 962 caregiver responses met the eligibility criteria. Patients reported SMA subtypes as: type I (25.0%), type I

SUBMITTER: McMillan HJ 

PROVIDER: S-EPMC8385498 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature