How to Administer Nusinersen in Adolescents 

How to Administer Nusinersen in Adolescents 

Nusinersen is the first treatment available for children with spinal muscular atrophy (SMA). Before its approval in 2016, the U.S. Food and Drug Administration had not approved the use of any drug for SMA treatment.1 While the introduction of this new drug marks...
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...
Respiratory Care in Spinal Muscular Atrophy: Intubation and Extubation 

Respiratory Care in Spinal Muscular Atrophy: Intubation and Extubation 

Intubation in spinal muscular atrophy (SMA) can be difficult or impossible,1 but the need for endotracheal intubation in SMA patients is increasing.2 In SMA type 1, respiratory failure can require frequent intubation in children ages three and below.3 As life...
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...
Respiratory Care in Spinal Muscular Atrophy: Airway Clearance

Respiratory Care in Spinal Muscular Atrophy: Airway Clearance

Weakness in the respiratory system is a common and serious complication of spinal muscular atrophy (SMA),1 and pulmonary disease is the primary cause of morbidity and mortality in SMA type 1 and SMA type 2 patients.2 Muscle atrophy and hypotonia are directly related...