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Setting realistic expectations for FA patients using Skyclarys

Sub Subramony, MD, shares how he communicates realistic expectations to his Friedreich’s ataxia patients regarding the benefits that Skyclarys could have for them.

About Sub Subramony, MD

Sub Subramony, MD, is a board-certified neurologist and neuromuscular medicine specialist at the Norman Fixel Institute for Neurological Diseases at University of Florida Health. He also serves as a professor in the University of Florida Department of Neurology with a joint appointment in pediatrics. His area of focus is genetic neuromuscular diseases, including Friedreich’s ataxia.

Transcript

Understand in the trial over a period of about 48 weeks — a yearlong trial almost — the patients who were treated with omaveloxolone were about 2.5 points in the so-called modified Friedreich’s ataxia rating scale, 2.5 points better than the ones that were on placebo.

This difference is not easily felt, I would say, by the patients. So I usually have a discussion with the patient saying that all the available evidence seems to suggest that Skyclarys seems to have a protective effect, it seems to slow down the progression. But you may not feel whether it is, you know, doing something.

Sadly, it’s unlikely that somebody who’s already in a wheelchair gets on this medication and then stands up and discards the wheelchair. I don’t anticipate that happening.

So like many other neuroprotective agents for other diseases that are approved, one has to kind of take it in blind faith that based on the trial results, this seems to have a protective effect.

And I would imagine that if the protective effect continues to function over the future course of the patient’s life, it will probably have an impact even more as the years go by in that if the patient is not taking this medication, he or she would have been much worse off 10 years down the line or 15 years down the line.

That will be my take on the medication right now. But nevertheless, patients have to accept this medication fully understanding that they may not suddenly see a quick recovery from some of the deficits they already have.

Our FA medical advisor

Dr. David LynchDavid Lynch, MD, PhD, is the director of the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia.

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