Why carry out this study?

The progressive, irreversible nature of spinal muscular atrophy (SMA) makes early diagnosis and uninterrupted care essential for optimal outcomes, particularly for the most severe form of the disease (Type 1).

Healthcare providers and SMA-affected individuals and their caregivers were surveyed to understand the effects of the COVID-19 pandemic on the continuity of clinical care and clinical trial conduct.

Respondents were asked which factors impacted SMA-related services that required in-person visits, such as trial recruitment, diagnosis confirmation, treatment administration, and physical therapy.

What was learned from the study?

Delays and cancellations of in-person SMA care and research appointments were common during the COVID-19 pandemic and were initiated by both healthcare providers and patients/caregivers to prevent viral exposure.

It is critically important that SMA healthcare providers and researchers develop contingency plans to avoid future disruptions in healthcare and clinical trial conduct.

Along with scheduling and logistical flexibility, priority should be placed on the development of remote clinical outcome assessments; local or mobile blood sample collection sites; remote physical therapy sessions; digital monitoring devices; and safe, on-schedule continuation of treatment administration.