Other therapeutic and supportive approaches for Friedreich's ataxia
Susan Perlman, MD, shares the therapeutic approaches she recommends for Friedreich’s ataxia patients outside of using Skyclarys (omaveloxolone).
About Susan Perlman, MD
Susan Perlman, MD, is a clinical professor of neurology and the director of the Ataxia Center at the UCLA Medical Center in Los Angeles. Her specialty is ataxias, including Friedreich’s ataxia. She is also the director of clinical trials in UCLA’s Program in Neurogenetics and sits on the Medical and Research Advisory Board at the National Ataxia Foundation.
Transcript
Once a diagnosis is made, we always get our patients involved with rehabilitation strategies — physical therapy, occupational therapy, speech therapy — when appropriate. If there are problems with swallowing, we can get nutrition involved or GI.
So, you know, I think, you know, using the resources that are used in any chronic neurologic disorder can improve quality of life. You know, orthotics for bracing, equipment consultations for walkers or wheelchairs — when or if those become necessary.
The use of off-label medications to help any of the ataxic symptoms, you know, over the years there have been a small number that have been brought out as being helpful to improve cerebellar function, reduce tremor, et cetera.
One is amantadine. Busprione has a literature supporting it and an equally large literature saying it doesn’t do much except lower anxiety levels. Riluzole has had some studies done in various ataxic illnesses, including Friedreich’s ataxia.
And then there are general tremor medications, medications that can be used for muscle cramping, medications that can be used for restless legs at night that are part of the neurologic armamentarium for those symptoms.
So there’s plenty we can do to improve comfort, improve sleep, and when appropriate, try medications that may improve balance.
David Lynch, MD, PhD, is the director of the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia.