I treated one of my first patients with myasthenia gravis (MG) in 1992. At the time, her weakness was so severe she had to be admitted to the intensive care unit. She was treated with amifampridine (Firdapse, Ruzurgi), which was considered an experimental therapy at the time. She referred to it as her “fire water,” and experienced a remarkable improvement.
Before treatments like these were available, about 70 percent of people died from their disease. Those with milder forms of MG often went undiagnosed. In the decades following the development of cholinesterase inhibitors like pyridostigmine (Mestinon, Regonol) in the 1930s, the risk of dying from MG gradually declined. By the 1980s, the mortality rate was less than 5 percent, and the number of people living with the disease was 1 in 17,000.
In the 30 years I've been practicing, I've cared for hundreds of patients with MG, many of whom live normal lives thanks to improvements in treatments and our understanding of the disease.
Despite these advances, a significant percentage of patients have continued to have symptoms that do not fully respond to treatment, and many more have side effects from medications used to treat MG such as prednisone. For these patients, the last several years have been exciting. The US Food and Drug Administration has approved four effective and innovative drugs that promise to revolutionize treatment. Numerous other medications are well along in clinical trials.
In our feature, you'll find more information about the various treatments for MG, including medication and surgery. Turn to our Healthy Living section to learn how exercise can help you manage your disease and how to do it safely. And since the COVID-19 pandemic isn't over yet, we spoke to a neuromuscular specialist on how to lower the risk of COVID-19 and whether vaccines are safe for people with MG.
As I reflect on the progress in the field of MG, my thoughts return to my patients over the years for whom available treatments didn't work adequately. I recall one man who was admitted to the intensive care unit about once a month despite whatever treatments we tried, and another who took almost a year to recover sufficient swallowing strength to allow him to eat independently. When I was a resident, I cared for a young woman with particularly severe MG who was admitted to the hospital several times per year. Her kindness and gratitude taught me and a generation of other neurology residents more about MG than any scientific paper.
I sometimes wonder what the future lives of these patients might have been like if the current treatments and others in the pipeline had been available then.
Looking ahead, I believe the stories in this issue will bring hope to people with MG, and fulfillment and joy to the teams of providers who care for them.
A. Gordon Smith, MD, FAAN, Issue Editor
