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

Case Report Shows Neuropathological, Molecular Findings of SMA Type 3 With Superficial Siderosis
November 28th 2020

Case Report Shows Neuropathological, Molecular Findings of SMA Type 3 With Superficial Siderosis

Jaime Rosenberg
Researchers have published their postmortem report on a case of spinal muscular atrophy type 3 complicated by superficial siderosis, offering details on their neuropathological and molecular findings.
Researchers Offer Indications of and Prophylactic Measures for Hepatotoxicity in SMA
November 26th 2020

Researchers Offer Indications of and Prophylactic Measures for Hepatotoxicity in SMA

Jaime Rosenberg
The compiled results revealed that hepatotoxicity related to the gene therapy typically presented as cholestatic, which could be prophylactically treated with prednisolone.
MRI room
November 21st 2020

Imaging Study Shows Upper Leg Involvement in SMA

Jaime Rosenberg
Using DIXON, T2 mapping, and diffusion tensor imaging, the researchers obtained quantitative MRI for 31 patients with spinal muscular atrophy (SMA) types 2 and 3.
Needle in vial
November 18th 2020

Nusinersen Improves Respiratory Muscle Strength in Patients With SMA Type 2

Jaime Rosenberg
Following 6 injections of nusinersen, respiratory muscle performance was significantly better among the treated patients compared with age‐matched historical controls.
Doctor holding baby
November 14th 2020

Using Nusinersen in Type 0 SMA Led to Mild Benefits, Case Study Shows

Jaime Rosenberg
At the time of treatment, there was just 1 paper documenting the use of nusinersen in a case of more mild type 0 spinal muscular atrophy(SMA).
DNA graphic
November 12th 2020

Researchers Identify Novel Mutations in SMA

Jaime Rosenberg
Researchers have identified additional mutations in both SMN1 and other genes, some of which may be associated with the onset of spinal muscular atrophy (SMA).
Neurons
November 7th 2020

Study Highlights Differences in Adaptive Skill Impairments Across NMDs

Jaime Rosenberg
The study emphasizes the importance of school support and other psychosocial interventions considering specific deficits across adaptive skills in patients with neuromuscular diseases (NMDs).
central nervous system graphic
November 6th 2020

Researchers Explore Respiratory Impact of Novel Therapies in SMA

Jaime Rosenberg
The researchers of the review discuss the reported impact of novel therapies for spinal muscular atrophy (SMA) and highlight the need for long-term data and monitoring of respiratory outcomes.
Stethoscope on money
October 30th 2020

Nusinersen Passes Cost-Effectiveness Thresholds for Infantile-Onset SMA

Jaime Rosenberg
The study offers the first de novo cost-effectiveness model of these patients in the United States, according to the researchers.
Newborn in parent hands
October 23rd 2020

Study Highlights Importance of Swift SMA Diagnosis Through Use of Newborn, Carrier Screening

Jaime Rosenberg
A new study is underscoring the importance of diagnostic testing for patients with spinal muscular atrophy (SMA) to ensure early diagnosis of the disease.
Image of brain
October 17th 2020

Analysis Shows Utility of Tool to Detect Motor Function Change in SMA

Jaime Rosenberg
Analyzing data from a phase 2 study of patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA,) researchers found that the assessment is a valid, reliable, and responsive assessment of motor function ability for these patients.
Picture of a survey
October 9th 2020

Survey Captures Baseline Experience of Patients With SMA

Allison Inserro
A recent study about the impact of spinal muscular atrophy (SMA) on patients and caregivers may help to illustrate the impact of future improvements as new therapies take hold, but more targeted SMA quality-of life-outcome measures may be needed to fully the changes.
Two-Year Risdiplam Data Show Continued Improvement in Motor Milestones in SMA
September 29th 2020

Two-Year Risdiplam Data Show Continued Improvement in Motor Milestones in SMA

Jaime Rosenberg
At the time of the 2-year analysis, 88% of the 17 infants who received the therapeutic dose of risdiplam for spinal muscular atrophy (SMA) were alive and did not require permanent ventilation.
Hip Instability Progresses Most Rapidly in Patients With SMA Type 1
September 27th 2020

Hip Instability Progresses Most Rapidly in Patients With SMA Type 1

Jaime Rosenberg
By the age of 4 years, all children with type 1 SMA included in the study had subluxated hips.
New Research Offers Key Safety, Early Efficacy Data on Zolgensma in SMA
September 25th 2020

New Research Offers Key Safety, Early Efficacy Data on Zolgensma in SMA

Jaime Rosenberg
A year after the FDA approved Zolgensma for the treatment of spinal muscular atrophy in children aged under 2 years without end-stage weakness, researchers shared safety and early efficacy data from the first 21 children treated with the gene therapy in Ohio.
Study Findings Offer More Understanding of Natural History of Type III SMA
September 17th 2020

Study Findings Offer More Understanding of Natural History of Type III SMA

Jaime Rosenberg
The study confirms previous observations of different trajectories between the progression of the 2 spinal muscular atrophy subtypes, the researchers said.
Higher Health Care Costs and Utilization for Infantile-Onset SMA
September 11th 2020

Higher Health Care Costs and Utilization for Infantile-Onset SMA

Laura Joszt, MA
Patients with spinal muscular atrophy (SMA) incur significant health care resource utilization and cost burden, particularly those with infantile-onset SMA.
Improved Treatment Options for SMA Present New Opportunities, Challenges
September 5th 2020

Improved Treatment Options for SMA Present New Opportunities, Challenges

Laura Joszt, MA
There are now multiple therapeutic options available for spinal muscular atrophy (SMA), a disease once considered incurable; however, there remains a need for additional treatments and better ways to predict treatment response.
Socioeconomic Status Impacted Reproductive Carrier Screening Testing in Australia
September 1st 2020

Socioeconomic Status Impacted Reproductive Carrier Screening Testing in Australia

Laura Joszt, MA
Reproductive carrier screening can identify conditions like spinal muscular atrophy, but this testing is often not done at all or not done before a woman gets pregnant, according to a new study.
Pediatrics
August 28th 2020

SMA Impacts Health-Related Quality of Life for Both Patients and Caregivers

Laura Joszt, MA
In addition to high health care and societal costs, spinal muscular atrophy (SMA) is associated with a deterioration in the health-related quality of life of both patients and their caregivers in 3 European countries.
Evrysdi logo
August 24th 2020

After FDA Approval, EMA Accepts Marketing Authorization Application for First Oral SMA Therapy

Laura Joszt, MA
The FDA approved the drug on August 7, and on August 17, the European Medicines Agency (EMA) accepted the marketing authorization application for the spinal muscular atrophy (SMA) treatment.
Need for Ventilation in Children With SMA Is Linked to Respiratory Function
October 2nd 2019

Need for Ventilation in Children With SMA Is Linked to Respiratory Function

Christina Mattina
Children with spinal muscular atrophy (SMA) who require noninvasive ventilation had more severe impairments in respiratory function than those who do not, according to findings of a new study.
Experts Discuss Symptoms, Types, and Treatments of SMA
September 22nd 2019

Experts Discuss Symptoms, Types, and Treatments of SMA

Christina Mattina
A recent Peer Exchange series from The American Journal of Managed Care® brought together a panel of experts to discuss spinal muscular atrophy (SMA), including its clinical presentation, diagnosis of its various types, and the potential of new disease-modifying treatments. The panel was moderated by Peter L. Salgo, MD.
FDA Approves Gene Therapy With $2.1M Price Tag for Spinal Muscular Atrophy in Pediatric Patients
May 24th 2019

FDA Approves Gene Therapy With $2.1M Price Tag for Spinal Muscular Atrophy in Pediatric Patients

Jaime Rosenberg
AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.
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