| 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. |