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...
Assistive Devices and Equipment: Oxygenation and Breathing 

Assistive Devices and Equipment: Oxygenation and Breathing 

Spinal muscular atrophy (SMA) is associated with serious respiratory system complications,1 and pulmonary disease is the primary cause of morbidity and mortality in both SMA type 1 and SMA type 2 patients.2 Given the frequency and extent of oxygenation and breathing...
Assistive Devices and Equipment in Spinal Muscular Atrophy: Braces

Assistive Devices and Equipment in Spinal Muscular Atrophy: Braces

Braces are one amongst a number of rehabilitation techniques that are used in those with neuromuscular disorders like spinal muscular atrophy (SMA).1 Their effectiveness is thought to vary depending on the individual case. Children with SMA may have spinal braces as...