Patient and Parent Perspectives on the Use of Nusinersen for Spinal Muscular Atrophy

Patient and Parent Perspectives on the Use of Nusinersen for Spinal Muscular Atrophy

At the end of 2016, the U.S. Food and Drug Administration (FDA) approved nusinersen for the treatment of spinal muscular atrophy (SMA).1 Because of the high threshold for FDA approval, an abundance of data on nusinersen and its physiological effects on SMA patients...
Spinal Muscular Atrophy: Awareness and Attitudes

Spinal Muscular Atrophy: Awareness and Attitudes

There is a paucity of information on awareness of and attitudes towards spinal muscular atrophy (SMA) and medical approaches to the disease.1,2 However, research into what is known about SMA has revealed that the public is largely unfamiliar with SMA, and even...
Nephrolithiasis (Kidney Stones) in Spinal Muscular Atrophy

Nephrolithiasis (Kidney Stones) in Spinal Muscular Atrophy

Nephrolithiasis is not a classic or well-documented symptom of spinal muscular atrophy (SMA). Nonetheless, anecdotal reports from physicians, patients, and caregivers suggest that patients with SMA may suffer from kidney stones more frequently than an otherwise...