Spinal Muscular Atrophy

Patients with spinal muscular atrophy (SMA) incur significant health care resource utilization and cost burden, particularly those with infantile-onset SMA.

There are now multiple therapeutic options available for spinal muscular atrophy (SMA), a disease once considered incurable; however, there remains a need for additional treatments and better ways to predict treatment response.

Reproductive carrier screening can identify conditions like spinal muscular atrophy, but this testing is often not done at all or not done before a woman gets pregnant, according to a new study.

In addition to high health care and societal costs, spinal muscular atrophy (SMA) is associated with a deterioration in the health-related quality of life of both patients and their caregivers in 3 European countries.

The FDA approved the drug on August 7, and on August 17, the European Medicines Agency (EMA) accepted the marketing authorization application for the spinal muscular atrophy (SMA) treatment.

Children with spinal muscular atrophy (SMA) who require noninvasive ventilation had more severe impairments in respiratory function than those who do not, according to findings of a new study.

A recent Peer Exchange series from The American Journal of Managed Care® brought together a panel of experts to discuss spinal muscular atrophy (SMA), including its clinical presentation, diagnosis of its various types, and the potential of new disease-modifying treatments. The panel was moderated by Peter L. Salgo, MD.

AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.