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...
What is Meaningful Change in Spinal Muscular Atrophy?

What is Meaningful Change in Spinal Muscular Atrophy?

A tool that is commonly used to evaluate motor function in spinal muscular atrophy (SMA) patients is the Hammersmith Functional Motor Scale (HFMSE).1 Given the extensive use of this tool, particularly for clinical trial research that aims to identify and develop...
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 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...
Pain Management in Spinal Muscular Atrophy

Pain Management in Spinal Muscular Atrophy

Increased pain is significantly associated with lower levels of health, social function, and vitality.1 The results of studies on animal models of neuropathic pain suggest that the abnormal excitability of dorsal horn neurons in spinal muscular atrophy (SMA) may lead...