Spinal Muscular Atrophy (SMA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Spinal muscular atrophy (SMA) is characterized by the degeneration of alpha motor neurons in the anterior horn cells of the spinal cord, leading to progressive weakness and wasting of proximal muscles and, in severe cases, paralysis. The clinical presentation of SMA varies from severe to mild. It is classified into three subtypes: type I (also known as Werdnig Hoffmann disease), type II, and type III (also referred to as Kugelberg Welander disease), as well as type IV. This condition involves muscle weakness and atrophy due to progressive degeneration and irreversible loss of lower motor neurons in the spinal cord and brain stem nuclei. The onset of weakness can occur from prenatal stages to adulthood, typically displaying a symmetric, proximal > or distal pattern that worsens over time. Previously, SMA was categorized into clinical subtypes based on the maximum motor function achieved; however, it is now understood that the phenotype of SMN1-associated SMA exists along a conti...