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...
Sleep Disorders in Spinal Muscular Atrophy

Sleep Disorders in Spinal Muscular Atrophy

Those with spinal muscular atrophy (SMA) often suffer from sleep disorders, but the specific reasons for sleep disturbances vary. For instance, some studies on SMA report sleep disturbances resulting from breathing disorders like sleep apneas, whereas others report...
Assistive Devices and Equipment in Spinal Muscular Atrophy: Feeding

Assistive Devices and Equipment in Spinal Muscular Atrophy: Feeding

Feeding is often a challenge in people with spinal muscular atrophy (SMA), particularly in those with more severe forms, and feeding difficulties can lead to malnutrition and suboptimal body composition.1,2 Loss of ability to sit is associated with the need for...
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...
Spinal Muscular Atrophy: Effects on Home, School, and Leisure

Spinal Muscular Atrophy: Effects on Home, School, and Leisure

Those with spinal muscular atrophy (SMA) face psychological and social challenges that often hinder quality of life.1 Even those who are able to live in their homes and participate in school, employment, and leisure activities are burdened with challenges related to...