On April 28, the US Food and Drug Administration (FDA) granted approval to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United . . .
Myotonic dystrophy (DM) is a type of muscular dystrophy that affects about 1 in 8,000 people worldwide. The disease is known by several names, including Steinert disease, after the doctor that first described it, and “DM” in reference to its Greek name, dystrophia myotonica. Similar to other forms of muscular dystrophy, DM is characterized by . . .
Duchenne muscular dystrophy (DMD) belongs to a group of rare genetic disorders, known as dystrophinopathies, that are caused by mutations of the dystrophin gene. DMD is characterized by progressive degeneration and weakness of the body’s voluntary muscles, primarily the skeletal muscles that control movement. In later stages, the heart and respiratory muscles may also be . . .
Located at more than 150 of the top healthcare institutions across the United States, MDA Care Centers serve as the nexus for expert clinical care and medical research. Our Care Center Network offers tens of thousands of appointments each year for individuals living with muscular dystrophy, ALS, and other neuromuscular diseases. These appointments provide individuals . . .