SMA August Roundup: green kiwifruit extracts, abnormal fatty acid metabolism & updates on nusinersen and AVXS-101

SMA August Roundup: green kiwifruit extracts, abnormal fatty acid metabolism & updates on nusinersen and AVXS-101

Summer is closing with an abundance of new data on SMA. Studies aimed at understanding the disease process continue to provide information on its underlying mechanisms. At the same time, careful investigations of therapeutics are highlighting the potential of these...
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: 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...
SMA Care Has Significant Financial, Career, Social, and Psychological Impact on Families, Study Reports

SMA Care Has Significant Financial, Career, Social, and Psychological Impact on Families, Study Reports

Taking care of a child with spinal muscular atrophy (SMA) significantly affects several aspects of  families’ lives, including finances, career choices, social life, and mental health, according to an Australian study. The study, “Financial, opportunity and...