Indications
Myasthenia Gravis
Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disease characterized by weakness and fatigue of voluntary muscles. The disease is caused by pathogenic antibodies that target the neuromuscular junction, including the acetylcholine receptor and the muscle specific tyrosine kinase receptor, disrupting normal function. We believe there are approximately 66,000 people in the United States and 104,000 people in Europe living with MG. MG is more prevalent in women and tends to affect younger women. In men, MG tends to affect an older population.
The clinical course of MG is variable but usually progressive. Symptoms typically emerge in the eyes (e.g., drooping eyes, double-vision, blurred vision) and progress into the face, throat, and limbs. The chronic nature of MG requires patients to cope with fluctuating symptoms and multiple treatment regimens for management. Overall, the persistent muscle weakness associated with MG often negatively interferes with patients’ ability to engage in daily and physical activities, including demands of work, family, and social functions.
Thyroid Eye Disease
Thyroid eye disease (TED) is an autoimmune disorder that affects the muscles and other tissues around the eyes. TED is most commonly caused by IgG autoantibodies that form against the thyroid-stimulating hormone receptor. These antibodies, which also cause Graves’ disease, activate certain cell types, such as fibroblasts and adipocytes, that are present in the extraocular space, promoting inflammation and cellular expansion that result in the clinical manifestations of the disease. We believe there are approximately 33,000 people in the United States and 52,000 people in Europe living with TED.
Approximately one in twenty patients with Graves’ disease will present with moderate-to-severe TED that is characterized by swelling and redness of the eyelids, proptosis (protrusion of the eyeball), double vision, and, in severe cases, corneal ulceration and decreased visual acuity.
Warm Autoimmune Hemolytic Anemia
Warm autoimmune hemolytic anemia (WAIHA) is a rare hematologic disease in which autoantibodies mediate hemolysis, or the destruction of red blood cells. The clinical presentation is variable and most commonly includes non-specific symptoms of anemia such as fatigue, weakness, skin paleness, and shortness of breath. Symptoms typically develop chronically over several weeks to months, but rapid progression over a span of days has also been observed. Based on published estimates, we believe that there are approximately 42,000 people in the United States and 67,000 people in Europe living with WAIHA.