Spinal Muscular Atrophy

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While the findings suggest that switching to onasemnogene abeparvovec after non–gene therapy treatments could be beneficial to patients with spinal muscular atrophy, it is not clear which patients may benefit the most from switching therapies. | Image credit: Rochu_2008 - stock.adobe.com
Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

August 15th 2025

Children with spinal muscular atrophy (SMA) showed motor function improvements after switching to onasemnogene abeparvovec following prior treatment with nusinersen or risdiplam in a real-world study.

FDA approval. | Image Credit: syahrir - stock.adobe.com
Risdiplam Tablet for Spinal Muscular Atrophy Receives FDA Approval

February 12th 2025

Top 5 SMA Articles 2024
Top 5 Most-Read SMA Articles of 2024

December 16th 2024

Depiction of SMA in the nervous system | image credit: janya - stock.adobe.com
Implications of SMN2 Copy Number: A Key Factor in SMA Outcomes?

December 12th 2024

Keeping track of subgroup variability in SMA will provide more robust, comparable data in the long term | image credit: Bartek - stock.adobe.com
Decoding SMA Progression: HFMSE Analysis Spotlights Variability

November 27th 2024

More News

Brain waves
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.
Intrathecal catheter
October 14th 2021

Subcutaneous Intrathecal Catheter Allows for Effective Administration of Nusinersen in SMA

Rose McNulty
A novel indwelling subcutaneous intrathecal catheter circumvented the complex spine anatomy of patients with spinal muscular atrophy (SMA), a challenge when giving nusinersen via intrathecal injection.
Stuffed animal and stethoscope
October 10th 2021

Despite Progress in SMA Identification and Treatment, More Work Remains

Rose McNulty
A review of advances in spinal muscular atrophy (SMA) identification and treatment highlights areas where there is room for improvement.
Blood sample in centerfuge
October 8th 2021

Serum Neurofilament Light Chain May Be a Viable Biomarker in SMA

Rose McNulty
Biomarkers for spinal muscular atrophy are needed to further analyze treatment efficacy, and a recent study points to serum neurofilament light chain levels as a possible option.
DNA section
October 3rd 2021

Modern Gene Therapies Show Promise in Motor Neuron Disorders

Rose McNulty
A review of current research and drug development in motor neuron disorders such as amyotrophic lateral sclerosis and spinal muscular atrophy shows progress in the identification of treatment pathways and therapies.
Child and parent at doctor's office
October 1st 2021

Assessing Upper-Limb Function in Neuromuscular Diseases: Current Tools and Needs

Rose McNulty
A review of instruments used to assess upper-limb function in patients with neuromuscular diseases found that more tools are needed to address specific diseases.
Gene therapy
September 26th 2021

Risk vs Impact: Viral Gene Therapy for SMA

Rose McNulty
A recent review of viral gene therapy in pediatric neurological diseases included positive outcomes in patients with spinal muscular atrophy (SMA).
Child in hospital
September 24th 2021

SMA Growth Patterns Differ From General Pediatric Percentiles

Rose McNulty
Recent research identified spinal muscular atrophy (SMA)–specific growth percentiles, aiming to improve clinical management of the nutritional aspects of the disease.
Picture of infant
September 18th 2021

Lack of Awareness, Screening Slows SMA Diagnosis in Infants

Rose McNulty
A pair of surveys found that there are knowledge and practice gaps preventing earlier diagnoses of spinal muscular atrophy (SMA) in infants and toddlers.
Photo of newborn child
September 16th 2021

Study Clarifies Prevalence and Incidence of SMA in Japan

Rose McNulty
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

Improving Outcomes-Based Managed Entry Agreements for SMA Therapy

Allison Inserro
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

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

Tyler Gamba
Even though respondents did not know very much about spinal muscular atrophy, the survey showed that most were in favor of newborn screening.
Neuron firing
March 27th 2021

Researchers Highlight Emerging Concepts Underlying Selective Neuromuscular Dysfunction in SMA

Jaime Rosenberg
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.
Close-up of microscope
March 25th 2021

Case Study Highlights Benefit of Nusinersen in Late-Onset SMA

Jaime Rosenberg
This case study, involving a boy aged 13 years, shows that in a little over 1 year, the treatment yielded significant clinical benefit.
microscope and dna model on a world map
March 19th 2021

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

Skylar Jeremias
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.
Young woman using wheelchair
March 14th 2021

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

Skylar Jeremias
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

Nusinersen Elicits Functional Innervation in Patients With SMA

Jaime Rosenberg
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.
Hands holding the world
March 12th 2021

SMA Costs Vary Widely Around the World by Disease Type

Larry Hanover
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.
Inpatient, Outpatient Costs of SMA Are “Tremendous,” Study Highlights
March 3rd 2021

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

Laura Joszt, MA
Direct inpatient and outpatient costs are significantly higher for patients with untreated spinal muscular atrophy (SMA) compared with matched controls.
Risdiplam Leads to Greater Production of Vital SMN, SMA Study Says
February 26th 2021

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

Allison Inserro
The results also showed that the infants achieved certain milestones in motor development, pulmonary function, and survival.
Language, Intellect, Speech Differ by Phenotype in Children With SMA Type 1
February 14th 2021

Language, Intellect, Speech Differ by Phenotype in Children With SMA Type 1

AJMC Staff
Speech impairment was primarily linked to motor impairment, according to a study examining the development trajectory of children with spinal muscular atrophy.
Emergence of Gene-Specific Therapies for SMA Brings Promise, Questions
January 27th 2021

Emergence of Gene-Specific Therapies for SMA Brings Promise, Questions

Jaime Rosenberg
While these therapies continue to revolutionize the treatment landscape of SMA, treatments are not without shortcomings or challenges.
SMA Associated With Metabolic, Nutritional Issues
January 24th 2021

SMA Associated With Metabolic, Nutritional Issues

Jaime Rosenberg
Documented issues include lipid metabolic abnormalities, glucose metabolic abnormalities, and altered vitamin levels.
Top 5 Most-Read SMA Stories of 2020
December 26th 2020

Top 5 Most-Read SMA Stories of 2020

Skylar Jeremias
The top 5 most-read stories about spinal muscular atrophy (SMA) of 2020 on AJMC.com focused on a new treatment option for SMA and whether gender or gene mutations have any effects on the disease.
What Are Pulmonary Function Testing Options in Neuromuscular Diseases Like SMA?
December 19th 2020

What Are Pulmonary Function Testing Options in Neuromuscular Diseases Like SMA?

Jaime Rosenberg
Researchers review pulmonary function testing measures that span 4 categories that can assess the respiratory muscle dysfunction that happens in diseases like spinal muscular atrophy (SMA).
Newly Studied Neurological Impact of Polio Could Have Implications for SMA
December 19th 2020

Newly Studied Neurological Impact of Polio Could Have Implications for SMA

Jaime Rosenberg
Researchers found abnormal coherence patterns in survivors of childhood polio.
Despite Favorable Safety, Olesoxime Falls Short Among Efficacy Markers for SMA
December 10th 2020

Despite Favorable Safety, Olesoxime Falls Short Among Efficacy Markers for SMA

Jaime Rosenberg
Long-term follow-up of the treatment showed that while it has a favorable safety profile, the treatment does not offer significant efficacy for patients with spinal muscular atrophy (SMA) type 2 and non-ambulant type 3.
Clinician reviewing results
December 7th 2020

Population Carrier Screening Increases Prenatal SMA Diagnoses, Reduces SMA Births

Jaime Rosenberg
Between the first introduction of population carrier screening in 2008 and its expansion to the full Israeli population in 2013, the mean rate of prenatal diagnoses per year was 4.66, and this rose to 7.75 between 2014 and 2017, a 66% increase.
Droplet of money
December 5th 2020

Patients With SMA Have Medical Costs Over 50 Times Higher Than Those of Healthy Patients

Jaime Rosenberg
For infants, the main driver of these increased costs came from inpatient visits vs outpatient visits among children and juveniles.
Close-up of eye
December 3rd 2020

Risdiplam Not Associated With Retinol Toxicity in SMA, Study Says

Jaime Rosenberg
Treatment with risdiplam may not elicit ophthalmologic toxicity in older patients, as well as infants with immature retinas.
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