Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability: Neurology Clinical Practice: Vol 14, No 1
Background and ObjectivesSpinal muscular atrophy (SMA) is a neurodegenerative disorder manifesting with progressive muscle weakness and atrophy. SMA type 1 used to be fatal within the first 2 years of life, but is now treatable with therapies targeting …