Spinal Muscular Atrophy

Study Findings Offer More Understanding of Natural History of Type III SMA

September 17th 2020, 10:00am

Article

The study confirms previous observations of different trajectories between the progression of the 2 spinal muscular atrophy subtypes, the researchers said.

Researchers Outline Promising Future for SMA, DMD With Emergence of Gene Therapies, ASOs

September 12th 2020, 2:00pm

Article

Researchers provided an overview of what they deem a hopeful future for spinal muscular atrophy and Duchenne muscular dystrophy.

Higher Health Care Costs and Utilization for Infantile-Onset SMA

September 11th 2020, 1:00pm

Article

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

Improved Treatment Options for SMA Present New Opportunities, Challenges

September 5th 2020, 3:30pm

Article

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.

Socioeconomic Status Impacted Reproductive Carrier Screening Testing in Australia

September 1st 2020, 12:00pm

Article

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.

SMA Impacts Health-Related Quality of Life for Both Patients and Caregivers

August 28th 2020, 7:25pm

Article

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.

After FDA Approval, EMA Accepts Marketing Authorization Application for First Oral SMA Therapy

August 24th 2020, 7:00pm

Article

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.

Need for Ventilation in Children With SMA Is Linked to Respiratory Function

October 2nd 2019, 8:30pm

Article

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.

Experts Discuss Symptoms, Types, and Treatments of SMA

September 22nd 2019, 5:00pm

Article

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.

FDA Approves Gene Therapy With $2.1M Price Tag for Spinal Muscular Atrophy in Pediatric Patients

May 24th 2019, 10:33pm

Article

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.