Spinal Muscular Atrophy

Latest News

Biomarker identification has the potential to transform screening, early identification and treatment for diseases like SMA | image credit: Катерина Євтехова - stock.adobe.com
Breaking: Novel Prognostic Biomarkers Discovered for SMA

October 9th 2024

CNTN1 and NRXN3 clearly differentiated severe and milder forms of spinal muscular atrophy (SMA).

Newborn screening can provide numerous benefits for infants who may have unknown conditions or complications | image credit: Peakstock - stock.adobe.com
5-Year Review Further Bolsters Support for Newborn SMA Screening

October 3rd 2024

SMA has previously been associated with multiple metabolic and nutritional issues | image credit: JEGAS RA - stock.adobe.com
New Research Unearths Evidence of Global Metabolic Disruption in Patients With SMA

September 21st 2024

Clinical decision-making in SMA could benefit from more comprehensive trial data comparing SMA treatments | image credit: HASAN - stock.adobe.com
Decision-Making in SMA Complicated by Insufficient Treatment Comparison

September 19th 2024

Nusinersen was the first FDA-approved drug for the treatment of SMA in pediatric and adult patients in December 2016 | image credit: AGPhotography - stock.adobe.come
Higher-Dose Nusinersen Meets Primary End Point in Phase 2/3 DEVOTE Study for Spinal Muscular Atrophy

September 17th 2024

Spinal Muscular Atrophy Spotlight

The introduction of 3 FDA-approved therapies for spinal muscular atrophy is enabling clinicians to treat the disease completely different from how it was handled in the past.

John Brandsema, MD of Children's Hospital of Philadelphia

More News

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.
<123456

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

© 2024 MJH Life Sciences® and AJMC®.
All rights reserved.

About
Contact
Editorial Boards
Advertise
Do Not Sell My Information
Terms & Conditions
Privacy Policy
AJMC Managed Markets Network Logo
CH LogoCenter for Biosimilars Logo