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Spinal Muscular Atrophy

Dennis P. Scanlon, PhD; Emma Ciafaloni, MD; Maria Lopes, MD, MS; Kevin U. Stephens, Sr, JD, MD; Mary Schroth, MD, FAAP, FCCP

Reducing Disease Burden Through Early Diagnosis and Treatment of Spinal Muscular Atrophy (SMA)

Hear an overview of the disease burden in spinal muscular atrophy and learn about clinical and payor perspectives on screening and diagnosis, current treatment options, and new disease-modifying therapies in SMA.

Lack of Awareness, Screening Slows SMA Diagnosis in Infants

September 18th 2021, 10:25am

Article

A pair of surveys found that there are knowledge and practice gaps preventing earlier diagnoses of spinal muscular atrophy (SMA) in infants and toddlers.

Study Clarifies Prevalence and Incidence of SMA in Japan

September 16th 2021, 6:20pm

Article

An epidemiological investigation of spinal muscular atrophy (SMA) in Japan provided clarity on the genetic condition’s prevalence and incidence in the country and highlighted the importance of newborn screening for SMA

Improving Outcomes-Based Managed Entry Agreements for SMA Therapy

September 12th 2021, 12:00am

Article

Innovative therapies for rare diseases such as spinal muscular atrophy (SMA), when paid for by public budgets in the European Union and elsewhere, are often managed by agreements between payers and drug companies, but details can be hard to discern.

Survey Shows Wide Support for Spinal Muscular Atrophy Screening in Japanese Newborns

September 3rd 2021, 11:00am

Article

Even though respondents did not know very much about spinal muscular atrophy, the survey showed that most were in favor of newborn screening.

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.