Video
Jill Jarecki, PhD, chief scientific officer at Cure SMA explained the current data and potential for gender to impact the severity spinal muscular atrophy (SMA).
Jill Jarecki, PhD, chief scientific officer at Cure SMA explained the current data and potential for gender to impact the severity spinal muscular atrophy (SMA).
Transcript
What are your thoughts regarding whether gender has any effects on disease severity?
Jarecki: So, I think that it's been discussed in our community a few times whether gender has any effects on disease severity. However, from our data that CureSMA, currently has, we see that SMA affects both sexes equally. Our patient reported database where we have over 8000 patient records and from our clinical database, we see no evidence that severity is based on sex.
Generally, our data shows a 50/50 split of male and female patients in the SMA community. However, it's possible that there are more nuanced differences in severity that we can't detect through our data at this point.