Spinal muscular atrophy (SMA) is a genetic disorder characterized by the progressive degeneration of motor neurons, leading to muscle weakness and atrophy. This condition primarily affects infants and children, though it can also manifest in adults. Recent advancements in medicine have brought transformative therapies to the forefront, offering hope to those affected by SMA. Treatments such as nusinersen, onasemnogene abeparvovec, and risdiplam target the underlying genetic causes of the disease, aiming to improve motor function and enhance quality of life. Understanding these innovative therapies and their mechanisms is essential for patients, families, and healthcare providers navigating the complexities of SMA management.
|
|
|
|
