by Dr. James F. Howard, Jr.

As a physician specializing in the treatment of myasthenia gravis (MG), a rare, debilitating neurological disorder, I witness on a daily basis the adversities my patients face. Imagine being a hard-working, active adult—a mom who drove her kids to school every day, who liked to garden and exercise—suddenly faced with a disease that makes it difficult to see, walk or even talk. This is the reality of MG. It steals muscle strength to the point where those living with the disorder can no longer do the things they love. But many fight back, and I am consistently inspired by the strength and perseverance my patients exhibit to overcome these challenges and to not let their disease define them.

June is an important month for the MG community because it marks Myasthenia Gravis Awareness Month—a time when the community can come together, share experiences, and increase awareness for this rare, debilitating disorder. For me, this month provides a key opportunity to leverage my experiences seeing and treating patients with MG to elevate the public’s understanding of the disease and call attention to the special challenges faced by those living with it, including the need for improved diagnostic approaches and new, effective treatment options.

 

Diagnosing and Treating MG

In the United States, a rare disease is one that affects fewer than 200,000 patients.1 While the number of patients affected may be small, the impact of rare diseases on patients, their families, and society is profound.

Like other rare diseases, people living with MG often face a long road to diagnosis since many physicians are unfamiliar with the signs and symptoms of MG. As a result, it may take patients several years before they are accurately diagnosed with MG and can begin treatment.2

Even then, there is no cure for MG, meaning people living with this disease cope with it over the course of their lives. While there are treatment options available that can help manage symptoms in many patients, some patients find that their disease is not adequately controlled with therapies. In fact, as many as 15 percent of MG patients have refractory MG—meaning that despite current treatment, they continue to suffer from debilitating muscle weakness that severely impairs their ability to engage in simple daily activities.3

 

Living with MG

Significant muscle weakness is the hallmark symptom of MG, but no two people experience MG exactly the same way. Basic functions that many take for granted—like chewing, swallowing and walking —become difficult. Other common symptoms include drooping eyelids, blurred vision, slurred speech, and weakness in the arms and legs. Between 15 and 20 percent of MG patients will experience a “myasthenic crisis”—difficulty breathing that can require hospitalization and may be life-threatening.4

While the physicality of this disorder is debilitating, the emotional strain MG patients experience can be just as devastating. Many MG patients feel isolated and worry they are losing their identity. Because of their symptoms, many have had to give up their careers and may feel like a burden to their families both financially and socially. Relationships and friendships can suffer since any day can bring debilitating symptoms that force patients to cancel plans. The ability to travel, or even take a quick trip to the grocery store, may diminish, further compounding feelings of isolation.

 

What’s Next for MG

For MG patients, learning to cope with a lifelong disease can be daunting, so it’s important to focus on cultivating a support system to help overcome challenges. Family, friends, fellow MG patients, and physicians all play a role in helping patients get through difficult times and cheering them on through the great times.

Today, I am optimistic about what the future holds for people living with MG. Unlike some rare diseases, MG is an active area of research, with new clinical trials under way. I am especially hopeful that new research may benefit patients with refractory MG—who continue to suffer without adequate treatment options.

My hope for the MG community is that the scientific advances over the past 30 years—and particularly today’s research—will lead to a better quality of life for patients with this disorder. Please join me today, and every day during the month of June, in celebrating Myasthenia Gravis Awareness Month and spreading the word about this rare disease.

For more information about MG, including education, support, and resources for people living with MG and their families, visit the Myasthenia Gravis Foundation of America website at www.myasthenia.org.

James F. Howard Jr., M.D., is a Distinguished Professor of Neuromuscular Disease and Professor of Neurology & Medicine at the University of North Carolina at Chapel Hill School of Medicine. 

 

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References

  1. National Organization for Rare Disorders. Resources & FAQs: https://rarediseases.org/for-patients-and-families/information-resources/resources-faqs/
  2. Conti-Fine BM, Milani M, Kaminski HJ. Myasthenia gravis: past, present, and future. J Clin Invest. 2006;116(11):2843-54.
  3. Suh J, Goldstein JM, Nowak RJ. Clinical Characteristics of Refractory Myasthenia Gravis. Yale Jour Biol Med. 2013;86: pp.255-260.
  4. Wendell LC, Levine JM. Myasthenia crisis. The Neurohospitalist. 2011;1(1):16-22.
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