Spinal Muscular Atrophy: Late-July Monthly Roundup

Spinal Muscular Atrophy: Late-July Monthly Roundup

In July, the literature pertaining to spinal muscular atrophy (SMA) has focused mainly on understanding the disease. Information on treatments has been published largely in review form, while there has also been some new information on managing the disease and what...
Spinal Muscular Atrophy: June Bi-Monthly Roundup

Spinal Muscular Atrophy: June Bi-Monthly Roundup

Since our last roundup, there has been continued focus in the spinal muscular atrophy (SMA) literature how best to treat the disease. There also continues to be research geared toward understanding the biology of the disease, as well as on SMA management, what it is...
Spinal Muscular Atrophy: Monthly Roundup

Spinal Muscular Atrophy: Monthly Roundup

Much of the most recent research regarding spinal muscular atrophy (SMA) has focused on understanding the mechanisms mediating the disease as well as the clinical value of different pharmaceutical interventions. There have been several reviews on the latter topic,...
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...