Spinal Muscular Atrophy

Researchers Highlight Emerging Concepts Underlying Selective Neuromuscular Dysfunction in SMA

March 27th 2021, 11:00am

Article

Conducting a review of data published over a 25-year period, researchers highlight the importance of this type of research they say offers insight that drives the success of SMA treatment.

Case Study Highlights Benefit of Nusinersen in Late-Onset SMA

March 25th 2021, 7:17pm

Article

This case study, involving a boy aged 13 years, shows that in a little over 1 year, the treatment yielded significant clinical benefit.

Poster Presentations Demonstrate New Safety and Efficacy Data on Zolgensma for SMA

March 19th 2021, 4:00pm

Article

Spinal muscular atrophy treatment Zolgensma was found to be safe and effective for long-term use and use in presymptomatic patients, according to posters presented at the 2021 MDA Virtual Clinical & Scientific Conference.

Study Offers Snapshot of Successes and Challenges of Teens and Young Adults With SMA

March 14th 2021, 12:30pm

Article

Results of a survey depicting the ways that spinal muscular atrophy (SMA) affects the lives of adolescents and young adults revealed areas of support that need to be addressed to ensure that patients are able to live their best possible lives.

Nusinersen Elicits Functional Innervation in Patients With SMA

March 14th 2021, 11:00am

Article

According to the researchers, the findings are paving the way toward precision medicine and may have broader use in tracking treatment response in adult-onset motor neuropathy in the future.

SMA Costs Vary Widely Around the World by Disease Type

March 12th 2021, 2:45pm

Article

Reliable data on the costs of spinal muscular atrophy (SMA) illness are scarce, leaving key questions unanswered as new, high-priced therapies continue to emerge.

Dr Vamshi Rao Discusses Possible Future Guidelines for SMA Care

March 7th 2021, 5:00am

Video

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago discusses how new treatments will influence future care guidelines for spinal muscular atrophy (SMA).

Inpatient, Outpatient Costs of SMA Are “Tremendous,” Study Highlights

March 3rd 2021, 8:58pm

Article

Direct inpatient and outpatient costs are significantly higher for patients with untreated spinal muscular atrophy (SMA) compared with matched controls.

Dr Vamshi Rao Discusses the Future of Combination Therapy in SMA

February 28th 2021, 12:45pm

Video

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses the future of combination therapies in the spinal muscular atrophy (SMA) space.

Risdiplam Leads to Greater Production of Vital SMN, SMA Study Says

February 26th 2021, 1:52pm

Article

The results also showed that the infants achieved certain milestones in motor development, pulmonary function, and survival.