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Diagnostic and therapeutic advancements have significantly curbed disease gravity
Myasthenia gravis is sometimes called the snowflake disease due to the variability in patient symptoms, severity and treatment. Onset of this autoimmune disorder may be sudden, with severe and generalized muscle weakness, or may be subtle. Fortunately, the ability to diagnose and treat myasthenia gravis has improved dramatically through the years — and this progress continues.
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“There are a lot of promising and strong treatments that are going to be available,” says Yuebing Li, MD, PhD, Head of the Myasthenia Gravis Program in Cleveland Clinic’s Neuromuscular Center. “For doctors, this is a very gratifying disease to treat because the patient will almost always get better.”
In the latest episode of Cleveland Clinic’s Neuro Pathways podcast, Dr. Li delves into current diagnostic and therapeutic guidelines for myasthenia gravis. He discusses:
Click the podcast player above to listen to the 21-minute episode now, or read on for a short edited excerpt. Check out more Neuro Pathways episodes at clevelandclinic.org/neuropodcast or wherever you get your podcasts.
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Dr. Li: I would call this the golden age for clinical research on myasthenia gravis. We have never experienced such a surge of new potential drugs and therapies for this disease.
There are many — probably at least five to seven — being studied right now as treatments, mostly for generalized myasthenia gravis. There are a few different classes of therapy. One is complement inhibitor therapy. Basically, the complement activation has been proven to be an integral part of myasthenia gravis. One of the FDA-approved products, eculizumab, is a complement inhibitor. It has been available since 2017 for the treatment of generalized myasthenia gravis. Due its cost and the fact that not all patients respond to it, eculizumab has been pretty much reserved for patients who have significant or refractory myasthenia gravis.
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Another class that is quite active are therapies that target the neonatal Fc receptor. Agents that act on this pathway basically try to block the recycling of immunoglobulin, including the antibodies for myasthenia gravis. Preliminary data suggest that this class is also fairly effective, but none of these therapies have yet been approved by the FDA [Note: Since this podcast was recorded, efgartigimod has been approved by the FDA for treatment of generalized myasthenia gravis.].
And there are other classes of immunotherapy being studied, so it’s quite an exciting time for patients with myasthenia gravis and the providers who take care of them.
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