Spinal Muscular Atrophy

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FDA approval. | Image Credit: syahrir - stock.adobe.com

Risdiplam Tablet for Spinal Muscular Atrophy Receives FDA Approval

February 12th 2025

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral solution.

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February 18th 2022

Global Assessment of Nusinersen Reimbursement Highlights Need for More Transparency

Jaime Rosenberg

The researchers investigated the role that economic evaluation and budget impact analysis plays in reimbursement decisions for nusinersen, collecting data from countries across the globe.

February 12th 2022

Researchers Determine What Patients, Caregivers Deem Meaningful Change in SMA Motor Function

Jaime Rosenberg

Through the use of patient testimonials as well as analysis of data from a study of patients with spinal muscular atrophy (SMA), researchers were able to describe patient-centered “meaningful outcomes” in the 32-item Motor Function Measure.

February 11th 2022

Early Detection Emphasized Amidst Era of Changing Clinical Course of SMA

Jaime Rosenberg

Coupled with these interventions for treating spinal muscular atrophy (SMA) is an emphasis on diagnosing the disease as early as possible to ensure optimal clinical outcomes for patients.

February 3rd 2022

Report Outlines Optimal Approaches to Address Nusinersen Treatment Gaps

Jaime Rosenberg

Researchers outlined recommended treatment plans to build back effective levels of nusinersen following treatment gaps.

Image of a scientist looking at a slide under a microscope

January 30th 2022

Risdiplam May Be Superior for Certain SMA Type, According to Indirect Comparison

Jaime Rosenberg

Researchers compiled data from studies of oral Evrysdi (risdiplam), Spinraza (nusinersen), and Zolgensma (onasemnogene abeparvovec), finding evidence that risdiplam may be a favorable alternative to nusinersen for type 1 SMA.

Image of pregnant woman holding ultrasound photos

January 27th 2022

Multidisciplinary Care Emphasized for Pregnant Women With SMA

Jaime Rosenberg

According to researchers, this case of a patient with spinal muscular atrophy (SMA) from Indonesia was the first to use a collaborative, multidisciplinary approach that involved obstetric, neurology, pediatric, and anesthesiology departments at a hospital.

January 25th 2022

FDA Gives Priority Review to Risdiplam in SMA for Infants Younger Than 2 Months

AJMC Staff

Risdiplam is approved for children older than 2 months with spinal muscular atrophy (SMA); an expanded approval would include the youngest infants before they show symptoms.

January 22nd 2022

Study: NGS May Be a Superior Method of Detecting SMN1 Gene Copy Number

Jaime Rosenberg

Among the 3 methods used across 478 samples included in the study, next-generation sequencing provided the most favorable results.

January 21st 2022

Estonia-Based Study Assesses Prevalence, Characteristics of SMA Diagnoses

Jaime Rosenberg

Over the 24-year period, there were 57 spinal muscular atrophy diagnoses in 53 families, and a disease birth prevalence of 1 per 8286 births in Estonia.

Image of scientist testing treatments in the lab

January 15th 2022

Non-SMN Approaches Offer Alternative, Supportive Strategies for SMA Treatment

Jaime Rosenberg

Outside of survival motor neuron (SMN) treatment, there are several alternatives used in cases where SMN approaches are not appropriate for treating spinal muscular atrophy (SMA).

January 11th 2022

Nusinersen Likely to Improve QOL for Patients With SMA

Jaime Rosenberg

Across most questionnaire domains, patients reported improvements to their quality of life (QOL) while receiving treatment for spinal muscular atrophy (SMA) with nusinersen.

January 7th 2022

Research Highlights Significantly Higher Risk of Mortality Among Patients With SMA Prior to Nusinersen Approval

Jaime Rosenberg

Based on the data from over 5000 patients with spinal muscular atrophy (SMA) and 50,000 age-matched controls, patients with SMA had a nearly 1.8-fold higher mortality rate.

January 5th 2022

Lesser Studied Nonrespiratory Complications in Nusinersen-Treated SMA1 May Affect Outcomes

Rose McNulty

The respiratory complications of spinal muscular atrophy are well-researched, but a new study provides insight into nonrespiratory complications that may be just as crucial.

December 23rd 2021

Case Series Gives Insight Into Surgical Intervention for SMA Patients with Severe Scoliosis

Rose McNulty

Patients with spinal muscular atrophy often present with severe scoliosis but face an elevated risk of complications from corrective surgery.

December 22nd 2021

Top 5 Most-Read Spinal Muscular Atrophy Stories of 2021

Skylar Jeremias

The top 5 most-read spinal muscular atrophy stories of 2021 on AJMC.com covered a variety of topics concerning the disease, including cost differences, the potential for combination therapies, and identifying biomarkers for treatment.

December 18th 2021

New Imaging Approach May Assess Disease Burden in Patients With SMA

Jaime Rosenberg

If further studies confirm a benefit by using Multispectral Optoacoustic Tomography (MSOT), the technology can help forge a path toward a noninvasive, bedside, nonionizing approach to early visualization of disease as well as evaluation of disease burden and disease progression in patients.

December 11th 2021

Scoliosis Surgery for Pediatric SMA1 Comes With Serious Complications, Study Says

Rose McNulty

A case series of corrective surgeries in children with severe scoliosis due to spinal muscular atrophy (SMA) type 1 showed a positive success rate, but with significant postoperative complications.

December 2nd 2021

Pilot Program Underscores Importance of Newborn Screening to Identify, Treat SMA

Rose McNulty

Testing for spinal muscular atrophy in an Australian newborn screening program identified patients across socioeconomic and cultural demographics, mitigating inequity and providing patients with access to multidisciplinary treatment.

November 26th 2021

Study Assesses Age-Related Sensory Neuropathy in SMA

Rose McNulty

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.

November 22nd 2021

More Research Needed on SMA-Associated Costs, Resource Use

Gianna Melillo

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

November 18th 2021

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

Rose McNulty

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

November 13th 2021

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

Rose McNulty

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.

November 11th 2021

Analysis of Nusinersen Shows Favorable Safety Profile in Adults With SMA

Rose McNulty

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.

November 6th 2021

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

Rose McNulty

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.

November 5th 2021

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

Rose McNulty

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.

October 30th 2021

Biomarkers for Adult SMA Patients Treated With Nusinersen Remain Elusive

Rose McNulty

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

October 30th 2021

Study Evaluates Potential Cerebrospinal Fluid Biomarkers in SMA Treatment

Rose McNulty

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.

October 23rd 2021

Motor Unit Number Estimation Provides Insight Into Nusinersen Effects in SMA

Rose McNulty

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

October 21st 2021

More SMA Biomarkers Are Needed to Gauge Disease Progression, Therapy Response

Rose McNulty

A review of current molecular and electrophysiological biomarkers in spinal muscular atrophy (SMA) concluded that more exploration is necessary to find noninvasive, yet accurate, measures of disease progression and therapy response.

October 16th 2021

New Study Clarifies Cognitive Function in Adults With SMA vs Healthy Adults

Rose McNulty

A recent study compared intelligence quotient test scores between adult patients with spinal muscular atrophy (SMA) and healthy adults.

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