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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.

Study Assesses Age-Related Sensory Neuropathy in SMA

November 26th 2021, 9:26pm

Article

Sensory nerve action potential amplitudes decreased as patients aged in a cohort of spinal muscular atrophy (SMA) type 1 patients compared with a healthy, age-matched control group.

More Research Needed on SMA-Associated Costs, Resource Use

November 22nd 2021, 9:56pm

Article

Authors of a systematic literature review concluded more research is needed to better understand the financial burden of spinal muscular atrophy (SMA).

Respiratory, Upper Limb Function Declines After Loss of Ambulation in SMA Type 3

November 18th 2021, 11:30pm

Article

A recent review provides insight into the clinical progression of treatment-naive patients with spinal muscular atrophy type 3 who have lost ambulation.

Real-world Data Find Nusinersen Safe and Effective in Patients With SMA1

November 13th 2021, 6:15pm

Article

A retrospective observational cohort study of patients ranging in age from infants to adults provides real-world evidence that nusinersen is safe and effective for those with SMA1.

Analysis of Nusinersen Shows Favorable Safety Profile in Adults With SMA

November 11th 2021, 7:15pm

Article

Data on nusinersen’s safety profile in adult patients are more limited than in infant and adolescent patients, but a recent study showed positive results based on cerebrospinal fluid and blood sample parameters.

Home Support Outweighs Nusinersen Delays in Patients With SMA During COVID-19 Lockdown

November 6th 2021, 2:00pm

Article

A small, single-center study determined that adequate support at home affected outcomes in motor ability more so than the first COVID-19 lockdown in Italy.

Nonadherence to Nusinersen Treatment Increases Health Care Utilization, Costs in SMA

November 5th 2021, 10:00pm

Article

Results of a retrospective claims database analysis suggest that adherence to scheduled intrathecal nusinersen injections may lead to improvements in comorbidities, health care utilization, and costs for spinal muscular atrophy (SMA) types 1, 2, and 3.

Biomarkers for Adult SMA Patients Treated With Nusinersen Remain Elusive

October 30th 2021, 10:30pm

Article

As spinal muscular atrophy (SMA) treatments advance, new biomarkers that correlate with clinical outcomes are needed to gauge treatment response.

Study Evaluates Potential Cerebrospinal Fluid Biomarkers in SMA Treatment

October 30th 2021, 12:43pm

Article

The single-center study explored cerebrospinal fluid neurofilament and protein levels in patients with spinal muscular atrophy (SMA) undergoing treatment with nusinersen in an effort to identify useful biomarkers as treatment options expand.

Motor Unit Number Estimation Provides Insight Into Nusinersen Effects in SMA

October 23rd 2021, 12:00pm

Article

The MScanFit method assessed changes in motor unit configuration in adult patients undergoing treatment for spinal muscular atrophy (SMA).