FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug...
What is Spinal Muscular Atrophy (SMA)?

What is Spinal Muscular Atrophy (SMA)?

  Spinal Muscular Atrophy (SMA) is a congenital neuromuscular disorder characterized most prominently by progressive muscle atrophy and weakness.1 SMA is a neurodegenrative disease and the weakness and loss of muscle tone associated with SMA are the direct result...
Spinal Muscular Atrophy: Prognosis

Spinal Muscular Atrophy: Prognosis

The disease course and prognosis of spinal muscular atrophy (SMA) depends heavily on the clinical classification in a particular patient. For example, the prognosis of children with SMA type 0 is very poor while the prognosis in SMA type 4 patients is relatively good....
Spinal Muscular Atrophy: Differential Diagnosis – Childhood Onset (> 6 months and older)

Spinal Muscular Atrophy: Differential Diagnosis – Childhood Onset (> 6 months and older)

Spinal Muscular Atrophy (SMA) that presents after 6 months of age will include the second most common presentation of SMA, type 2 SMA, and the least commonly observed form of childhood onset SMA, type 3.1 The typical presenting symptoms of types 2 and 3 SMA,...
Orthopedic Surgery – Chest Deformity, Hip Instability, Contractures, and Fractures

Orthopedic Surgery – Chest Deformity, Hip Instability, Contractures, and Fractures

Children with spinal muscular atrophy (SMA) suffer from a number of orthopedic conditions. Scoliosis of the spine is quite common and is usually treated with bracing and/or instrumentation. However patients with SMA also experienced chest deformities that can lead to...