Video
Raising Awareness for Rare Diseases, Myasthenia Gravis: Hong Sun, MD, PhD
Author(s):
The global compound development team leader at Janssen discussed the importance of Rare Disease Day and one in particular, myasthenia gravis, which affects approximately 36,000 to 60,000 people in the US. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"Despite the available standard of care for patients, there’s still significant unmet need because chronic use of corticosteroids and suppressants usually are associated with significant [adverse] effects. About 28% of patients don’t respond to the standard of care treatment.”
On February 28, communities across the globe come together to celebrate Rare Disease Day, a day of recognition and raising awareness for the 300 million people worldwide living with a rare disease. A disease is defined as rare if it affects less than 200,000 people in the US. Despite this, there are more than 8000 rare diseases, of which many have neurological manifestations. Furthermore, about 80% of rare diseases are genetically based, and onset often occurs in childhood.1
Myasthenia gravis (MG), a rare chronic autoimmune disease, has an incidence rate of approximately 36,000 to 60,000 cases in the United States. This disease causes weakness in the skeletal muscles that worsens after periods of activity and improves after periods of rest. It typically occurs in women ages 20-40 years and men ages 50-80 years, but it can present at any age.2 Although MG is rarely seen in infants, the fetus may acquire antibodies from a mother affected with MG—a condition called neonatal myasthenia.
Today, MG can generally be controlled. There are several therapies approved to help reduce and improve muscle weakness. One investigational agent, nipocalimab (Janssen), has shown promising results in phase 2 settings and is currently being evaluated in a phase 3 trial. Hong Sun, MD, PhD, global compound development team leader at Janssen, sat down with NeurologyLive® to discuss the critical need to observe these rare diseases along with the day-to-day life a patient with MG typically experiences.
REFERENCES
1. Rare diseases at FDA. FDA. https://www.fda.gov/patients/rare-diseases-fda. Updated February 20, 2022. Accessed February 27, 2022.
2. Myasthenia gravis fact sheet. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Myasthenia-Gravis-Fact-Sheet. Updated November 15, 2021. Accessed February 27, 2022.
