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By the age of 4 years, all children with type 1 SMA included in the study had subluxated hips.

 Hip Instability Progresses Most Rapidly in Patients With SMA Type 1

A year after the FDA approved Zolgensma for the treatment of spinal muscular atrophy in children aged under 2 years without end-stage weakness, researchers shared safety and early efficacy data from the first 21 children treated with the gene therapy in Ohio.

New Research Offers Key Safety, Early Efficacy Data on Zolgensma in SMA

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Multiple treatment options allow better access to care and more health care opportunities for patients with spinal muscular atrophy (SMA), explained Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA.

Dr Mary Schroth Discusses How SMA Treatments Have Changed Patients' Lives

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

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

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

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

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

Higher Health Care Costs and Utilization for 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.

Improved Treatment Options for SMA Present New Opportunities, Challenges

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.

Socioeconomic Status Impacted Reproductive Carrier Screening Testing in Australia

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.

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

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.

Spinal Muscular Atrophy

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