Dr Mary Schroth Discusses At-Home Treatment Options for Patients With SMA

The latest FDA approved treatment is Evrysdi, which is an oral at-home therapy. What are the benefits of having this additional therapy available?

The FDA approval of Evrydi (risdiplam) plan provides another treatment option for people living with SMA. The ability to take a medication at home and by mouth or feeding tube will allow more people access to an SMA treatment. With multiple treatments for us to may available, patients should work in consultation with their health care provider team to determine which treatment option is best for their specific needs.

How has the COVID-19 pandemic affected people with SMA and their caregivers? For instance, are they more at risk of getting infected? Do we know what impact the virus has if they do get COVID-19?

Per feedback from our community about COVID-19, we learned that COVID-19 temporarily interfered with patients being able to receive treatments as scheduled. Monitory care transitioned to more telemedicine delivery and providing care in-person when necessary. Similarly, clinical trial visits have been modified to conduct in-person visits when essential, and other visits were modified to be conducted virtually.

We have limited reports of people living with us may have actually having COVID-19. In general, people living with SMA avoid others who are sick. And during the winter viral season, they already were avoiding public contact as much as possible. There's not enough information available to make any conclusions about the impact of infection on those with SMA. We surveyed our SMA community and we learned that less than 5% actually had COVID-19 infection or symptoms of COVID-19.