October 13, 2020Journal of Neuromuscular DiseasesOpen Access

The Cure SMA Membership Surveys: Highlights of Key Demographic and Clinical Characteristics of Individuals with Spinal Muscular Atrophy

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Why the study?

Annual surveys were initiated by Cure SMA to collect demographic, disease characteristics, healthcare, and burden of disease information directly from individuals with SMA and their caregivers.

Population

695 individuals with SMA in 2017 and 760 individuals in 2018

Design

Annual membership survey

Authors

LBLisa BelterUnited States Department of Commerce JJJill JareckiCure Spinal Muscular Atrophy SRSandra P. ReynaNovartis (Switzerland)

Discussion

Member takes

Implication

Survey data benchmark SMA type I timelines; leaves open effects of newer therapies on diagnosis and survival.

Structured PICO

Population

Individuals with spinal muscular atrophy (SMA) and their caregivers (n=1,142 unique individuals across 2017 and 2018 surveys)

Intervention

Annual patient-reported surveys (Cure SMA membership surveys)

Outcome

Demographic and disease characteristics, healthcare, and burden of disease informationpatient reported

The Cure SMA surveys provide valuable patient-reported demographic and clinical data that align with expected SMA population characteristics, aiding in advocacy and healthcare planning.

Cite This Study

Belter et al. (2020) studied this question.

synapsesocial.com/papers/6a84fe867a820f5775990bb4 https://doi.org/10.3233/jnd-200563

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