Screening Friedreich's ataxia patients for Skyclarys treatment
George Wilmot, MD, PhD, shares the demographics, such as age and disease stage, that make for ideal candidates for treatment with Skyclarys.
About George Wilmot, MD, PhD
George Wilmot, MD, PhD, is a board-certified neurologist affiliated with a few hospitals, including Emory University Hospital. He also serves as an associate professor at Emory University School of Medicine’s Department of Neurology and adjunct associate professor at the the school’s Department of Pediatrics. He is an ataxia clinical researcher and he started the Cooperative Ataxia Registry.
Transcript
Well, Skyclarys is FDA-approved for 16 and over. And, so that’s the appropriate age.
The drug has been shown to perhaps reduce the progression rate to some degree. And in doing that, I think that it can be an important thing for providers to prescribe for the right patients.
I think most Friedrich’s patients that fit in the right age group that don’t have contraindications — like bad liver failure, bad heart failure — those really weren’t studied in the studies that led to its approval; they were excluded. So most other patients, I think, have the potential for benefit.
I’ve had patients that have declined taking it because they are at such a late stage that it really didn’t make sense for them to prolong their disease. For that reason, you know, the very, very late-stage Friedrich’s is a question of whether they should take it or not.
But I think the majority of patients that are of appropriate age are possible candidates and really should be considered for Skyclarys therapy.
David Lynch, MD, PhD, is the director of the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia.