Spinal Muscular Atrophy

Latest News

Children with SMA are commonly affected by low bone mineral density | image credit: PT - stock.adobe.com
Low Bone Mineral Density Risk Increases With Age in SMA

November 13th 2024

Investigators used 3-dimensional quantitative computed tomography to more accurately assess patients’ bone mineral density.

Patients with SMA and their caregivers stand to benefit from shared decision-making processes | image credit: Intelligent Horizons - stock.adobe.com
Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments

November 8th 2024

SMA type is a strong predictor of some oral functioning in affected patients | image credit: RTimages - stock.adobe.com
Oral Function Impairment Varies Across Adult Patients With SMA

October 28th 2024

SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com
Protein Level Correlates With Pediatric SMA Severity, Treatment Response

October 17th 2024

SMA puzzle pieces | image credit: Dzmitry - stock.adobe.com
Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA

October 17th 2024

More News

genetics graphics
August 16th 2022

First Known Case of Rare Immunological Syndrome After SMA Gene Therapy Reported

Jaime Rosenberg
The case describes hemophagocytic lymphohistiocytosis (HLH), which occurred in a young boy in Italy with spinal muscular atrophy, who was treated with onasemnogene abeparvovec.
Pediatric medicine
August 6th 2022

New ANCHOVY Trial Data Highlight Benefits of SMA Therapy

Jared Kaltwasser
New data show the stark contrast between untreated infants with type 1 spinal muscular atrophy (SMA) and those who received risdiplam.
Image of a newborn
August 5th 2022

Japanese SMA Screening Program Could Help Speed Therapy

Jared Kaltwasser
Still, investigators say there are gaps in spinal muscular atrophy (SMA) screening protocol that need to be filled.
Image of silhouetted children
July 29th 2022

Nusinersen Leads to Broad Improvement in Children, Teens With SMA in Poland

Jared Kaltwasser
The study also found broad tolerability, with 98% of patients remaining on the therapy after 12 months.
Image of a doctor-patient consult
July 26th 2022

Mild SMA Poses Diagnostic Difficulties for Clinicians

Jared Kaltwasser
A new case report highlights the difficulties in pinning down spinal muscular atrophy (SMA) when it presents in a mild form.
picture of leg and foot
July 21st 2022

Muscle Ultrasound Could Be Important Tool in Flagging Late-Onset SMA

Jared Kaltwasser
The technique outperformed physical examinations in identifying muscle fasciculations, which are common in spinal muscular atrophy (SMA).
Image of a patient
July 18th 2022

Therapy Targeting Neuromuscular Junction Leads to SMA Improvement

Jared Kaltwasser
New data suggest motor function improves with amifampridine.
Image of a spine
July 14th 2022

Physical Therapy Boosts Efficacy of Nusinersen in SMA, Study Shows

Jared Kaltwasser
Patients who received regular physical therapy had improvement that was 4 times better than nusinersen alone, findings from this new study show.
Image of patient costs
July 11th 2022

Early Identification of SMA May Help Curb Costs of Disease

Jaime Rosenberg
When excluding the price tag of treatment, costs were significantly lower among the patients identified by early screening than among the patients identified by symptoms
Image of a newborn
July 2nd 2022

Initiation of Nusinersen for SMA Soon After Birth May Allow for Normal Outcomes

Jaime Rosenberg
Publishing their findings on the earliest treated infants to date, the researchers detailed the outcomes of 2 patients treated with nusinersen in their first week of life after receiving a spinal muscular atrophy (SMA) prenatal diagnosis.
Image of blood and DNA
June 29th 2022

Comprehensive Analysis of SMA May Improve Detection, Lower Residual Risk Ratio

Jaime Rosenberg
The researchers of this new study wrote that although SMA carrier screening is recommended for all couples, current testing methods primarily focus on copy number loss of SMN1, which can result in false-negative test results due to intragenic mutations and silent carriers falling under the radar.
Image of a spine x-ray
June 23rd 2022

Reduced Vertebral Body Height, Depth May Influence Surgical Intervention of SMA, Says Study

Jaime Rosenberg
The study included children and adolescents, and it found decreased vertebral height and depth among both groups of patients compared with healthy controls.
Patients With Calf Muscle Weakness Should Have SMA Gene Analysis, Study Says
June 17th 2022

Patients With Calf Muscle Weakness Should Have SMA Gene Analysis, Study Says

Jaime Rosenberg
Based on their findings, the researchers urged for the inclusion of a VRK1 gene analysis in the diagnostic workup of patients who present with predominant calf muscle weakness.
Image of a Black woman on a laptop
June 10th 2022

Patient Survey Findings Show Positive Attitudes Toward SMA Trial Experiences

Jaime Rosenberg
The results, say the study researchers, offer new insights into patient experiences during spinal muscular atrophy (SMA) clinical trials
mother and child meeting with doctor
June 8th 2022

Assessing Cost and QOL in Patients With SMA Before Symptoms Appear

Laura Joszt, MA
Identifying patients with spinal muscular atrophy (SMA) early and before the onset of symptoms can yield financial savings and improve quality of life (QOL) for these individuals and their families.
Risdiplam Expanded to Include Presymptomatic Infants Under 2 Months Old
June 3rd 2022

Risdiplam Expanded to Include Presymptomatic Infants Under 2 Months Old

AJMC Staff
The label expansion means risdiplam is approved for spinal muscular atrophy for all ages.
Image of genetic screening
March 26th 2022

Incorporating Risk Modifier Into Screening May Identify More Potential SMA Carriers

Jaime Rosenberg
Although most SMA carriers can be identified through routine SMN1 copy number assessment data, approximately 4% of carriers who harbor 2 copies of SMN1 can go undetected.
Image of a red medical cross
March 19th 2022

Initial RCT Data Support Safety, Efficacy of Nusinersen in SMA

Jaime Rosenberg
Across the studies of a new analysis, there was a significant risk difference in motor milestone response and improvement in Hammersmith Infant Neurological Examination score.
picture of blood droplet
March 17th 2022

Skeletal Muscle Biomarkers Altered in Type of SMA, Study Says

Jaime Rosenberg
In the majority of the 49 patients with a type of spinal muscular atrophy called spinal muscular atrophy Jokela, creatine kinase was elevated, particularly in males.
Young person using wheelchair
March 12th 2022

Researchers Outline Management Strategies for Growing Adult SMA Population

Jaime Rosenberg
Due to advances in the treatment of spinal muscular atrophy (SMA), the lifespan of patients with the disease has increased significantly, allowing them to enter adulthood. However, the change in SMA prognosis has also led to a knowledge gap in the natural history, management, and treatment strategy for adult patients, according to the researchers.
Image of a person pondering a decision
March 10th 2022

Shifting SMA Landscape Poses Ethical Considerations for Screening, Treatment

Jaime Rosenberg
Across these ethical considerations, the researchers emphasize the importance of collaborating with patients and their caregivers.
Image of clinical research
March 5th 2022

Researchers Urge Incorporation of Multidimensional Fatigue Scale in SMA Care

Jaime Rosenberg
The researchers observed internal consistence and convergent validity, leading them to suggest the tool be incorporated as a clinical standard in spinal muscular atrophy (SMA) care.
lung graphic
March 4th 2022

PEF May Be Preferred Measure of Respiratory Strength for SMA Follow-Up

Jaime Rosenberg
With clear differences at baseline and subsequent declines seen in a linear pattern, the researchers suggested peak expiratory flow may be a suitable outcome measure for longitudinal assessment of respiratory muscle strength in these patients.
Child in hospital bed
February 24th 2022

Study: Nusinersen Continues to Improve Motor Function in SMA in Second Year of Treatment

Jaime Rosenberg
Improvements, explained the researchers, were mainly driven by younger patients, with smaller improvements seen in older patients.
Image of the human body
February 19th 2022

Research Confirms Common Feature of Type 2 SMA, Reveals Novel Insights

Jaime Rosenberg
According to researchers, their findings warrant close surveillance of weight and BMI/age z-scores for patients with type 2 spinal muscular atrophy (SMA).
globes
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.
Teddy bear and stethoscope
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.
Image of scientist doing research
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.
doctor coat
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.
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