Spinal Muscular Atrophy

Latest News
February 12th 2025

Risdiplam Tablet for Spinal Muscular Atrophy Receives FDA Approval

December 16th 2024

Top 5 Most-Read SMA Articles of 2024

December 12th 2024

Implications of SMN2 Copy Number: A Key Factor in SMA Outcomes?

November 27th 2024

Decoding SMA Progression: HFMSE Analysis Spotlights Variability

November 21st 2024

Neuronal Deficit in Severe Infantile SMA Broader Than Previously Thought, Study Finds

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March 7th 2021

Dr Vamshi Rao Discusses Possible Future Guidelines for SMA Care

February 28th 2021

Dr Vamshi Rao Discusses the Future of Combination Therapy in SMA

February 14th 2021

Dr Vamshi Rao Discusses Safety, Efficacy of SMA Treatments

February 7th 2021

Dr Vamshi Rao Discusses How COVID-19 Impacted SMA Treatment Access

January 31st 2021

Dr Vamshi Rao Discusses How New Therapies Are Changing Physician Perceptions on SMA Prognosis

October 31st 2020

Dr Jill Jarecki Discusses Whether Gender Affects SMA Severity

October 25th 2020

Dr Jill Jarecki Discusses the Early SMA Treatment for Infants

October 18th 2020

Drs Mary Schroth and Jill Jarecki Discuss Treatments for All SMA Types

October 11th 2020

Dr Jill Jarecki Discusses the Current Pipeline for SMA Therapies

October 4th 2020

Dr Mary Schroth Discusses At-Home Treatment Options for Patients With SMA

More News

Children with SMA are commonly affected by low bone mineral density | image credit: PT - stock.adobe.com
November 13th 2024

Low Bone Mineral Density Risk Increases With Age in SMA

Jared Kaltwasser
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
November 8th 2024

Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments

Karen Jacobson-Sive
Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.
SMA type is a strong predictor of some oral functioning in affected patients | image credit: RTimages - stock.adobe.com
October 28th 2024

Oral Function Impairment Varies Across Adult Patients With SMA

Karen Jacobson-Sive
Oral function should be measured routinely to determine individual bulbar involvement stages in spinal muscular atrophy (SMA).
SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com
October 17th 2024

Protein Level Correlates With Pediatric SMA Severity, Treatment Response

Jared Kaltwasser
Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels of the protein increased after therapy.
SMA puzzle pieces | image credit: Dzmitry - stock.adobe.com
October 17th 2024

Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA

Marco Meglio
Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for US and EU regulatory submissions in Q1 2025.
Biomarker identification has the potential to transform screening, early identification and treatment for diseases like SMA | image credit: Катерина Євтехова - stock.adobe.com
October 9th 2024

Novel Prognostic Biomarkers Discovered for SMA

Karen Jacobson-Sive
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
October 3rd 2024

5-Year Review Further Bolsters Support for Newborn SMA Screening

Kyle Munz
A retrospective analysis on newborn screening for spinal muscular atrophy (SMA) provides further evidence for the benefits of early identification and treatment of the disease.
SMA has previously been associated with multiple metabolic and nutritional issues | image credit: JEGAS RA - stock.adobe.com
September 21st 2024

New Research Unearths Evidence of Global Metabolic Disruption in Patients With SMA

Kyle Munz
A recent analysis investigated the state of metabolic disruption experienced by patients with spinal muscular atrophy (SMA), adding to the literature on nutritional and metabolic complications linked to SMA.
Clinical decision-making in SMA could benefit from more comprehensive trial data comparing SMA treatments | image credit: HASAN - stock.adobe.com
September 19th 2024

Decision-Making in SMA Complicated by Insufficient Treatment Comparison

Nameera Temkar, MD
Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment options.
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
September 17th 2024

Higher-Dose Nusinersen Meets Primary End Point in Phase 2/3 DEVOTE Study for Spinal Muscular Atrophy

Isabella Ciccone, MPH
Based on the positive topline data, the company plans to submit for regulatory approval for a higher dose of nusinersen for spinal muscular atrophy (SMA) treatment.
The 6-Minute Walking Test is a common tool for measuring fatiguability in SMA | image credit: Antonioguillem - stock.adobe.com
August 28th 2024

Nusinersen May Improve Fatiguability in SMA, but More Research Is Needed

Kyle Munz
Findings from an Italian cohort suggest that nusinersen may benefit fatigue measures in patients with spinal muscular atrophy (SMA) type III; however, drawing definitive conclusions in this area remains difficult.
The SMN protein is expressed thoughout many body tissues types, including the liver | image credit: Rasi - stock.adobe.com
August 22nd 2024

SMN Depletion May Increase Risk of Liver Damage, Fatty Liver in SMA

Kyle Munz
A retrospective, single-center cohort study utilized liver ultrasound, serum collection, and proteome analysis to draw associations between spinal motor neuron (SMN) protein depletion and heightened risk for fatty liver disease in spinal muscular atrophy (SMA).
VR can reduce fear and anxiety associated with drawing blood in patients with SMA | image credit: matrosovv - stock.adobe.com
August 21st 2024

Virtual Reality Is a Game-Changer During Blood Draws for Pediatric Patients With SMA

Abraham Homer, MS
In honor of Spinal Muscular Atrophy (SMA) Awareness Month, Abraham Homer, MS, the gaming technology supervisor and creative technologist at Children's Hospital Colorado, shared how virtual reality transformed care for patients with SMA.
Apitegromab administered at 20mg/kg every 4 weeks lead to improvements in motor functions in patients with SMA | image credit: Justlight - stock.adobe.com
August 5th 2024

New Motor Milestones Achieved or Sustained With Apitegromab in Nonambulatory SMA

Kyle Munz
The extension of the TOPAZ study provided support for the long-term benefits and safety of apitegromab therapy in patients with spinal muscular atrophy (SMA) at 36 months.
SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com
July 26th 2024

Potential Biomarkers Predict Disease Severity and Treatment Response in SMA

Kyle Munz
Results from a single-center, observational, prospective study suggested that inflammatory markers could predict disease severity and treatment efficacy for patients with spinal muscular atrophy (SMA).
Globally, SMA is diagnosed for an estimated 1 in every 10,000 births | image credit: Alexander Raths - stock.adobe.com
July 19th 2024

SMA Birth Prevalence in US Lower Than Expected, New Report Finds

Kyle Munz
A new report provided updated figures estimated the birth prevalence of spinal muscular atrophy (SMA) in the US.
Onasemnogene abeparvovec is a gene therapy approved in the last decade for SMA | image credit: ipopba - stock.adobe.com
Published: July 12th 2024 | Updated: July 12th 2024

Dual Therapy Well-Tolerated but Presents Little Benefit to Infantile-Onset SMA

Kyle Munz
A multicenter pilot study concluded that dual therapy in spinal muscular atrophy (SMA) does not provide further benefits to infants at risk for SMA type 1.
Risdiplam demonstrated great 5-year efficacy for improving motor functioning and feeding capabilities in children with type I SMA | image credit: Sittipol - stock.adobe.com
July 3rd 2024

Final Readout of FIREFISH Study Showcases Risdiplam’s Pronounced Effects in Spinal Muscular Atrophy

Marco Meglio
The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam.
Medical Caregiver Concept | image credit: Alexis Scholtz - stock.adobe.com
December 31st 2023

Qualitative Study Explores Caregiver Experiences in SMA Treatment

Kyle Munz
This qualitative study conducted interviews with caregivers of patients with spinal muscular atrophy (SMA) to explore their unique experiences and challenges in navigating health systems to access disease-modifying therapies.
hospital | Image credit: VILevi - stock.adobe.com
December 27th 2023

More-Expensive SMA Medication Use Led to Lower Health Care Resource Utilization

Skylar Jeremias
Patients with spinal muscular atrophy (SMA) treated with a more expensive medication were found to have higher pharmacy costs but lower SMA-related health care resource utilization and medical costs compared with patients receiving standard-of-care nusinersen monotherapy.
Heavier Baby | image credit: Leka - stock.adobe.com
December 26th 2023

Onasemnogene Abeparvovec Effective in Treatment of Older, Heavier Children with SMA

Kyle Munz
A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1.
Lumbar Region Model | image credit: Crystal light - stock.adobe.com
December 20th 2023

Alternative Method of Nusinersen Administration Identified for SMA Cases With Spinal Deformities

Kyle Munz
A retrospective, single-center study out of the Czech Republic tested the safety and efficacy of unilateral interlaminar fenestration (UILF) on the convex side in cases of SMA that also exhibit severe spinal deformities.
Top 5 SMA Content 2023
December 16th 2023

Top 5 Most-Read Spinal Muscular Atrophy Content 2023

Kyle Munz
This year’s most-read spinal muscular atrophy(SMA) articles explored mental health, new technologies, caregiver well-being, and more.
Acetaminophen Bottle | image credit: luchschenF - stock.adobe.com
December 6th 2023

Acetaminophen Use Requires Careful Dosing in Patients With SMA, Study Says

Kyle Munz
A non-randomized, open-label, single-site clinical trial evaluated the safety and pharmacokinetics of acetaminophen intake in adults and children with spinal muscular atrophy (SMA) type II.
Cognitive Impairment Model | image credit: Pol Solé - stock.adobe.com
November 30th 2023

SMA May Be Linked to Cognitive Impairment in Chinese Patients

Kyle Munz
A cross-sectional study suggests that spinal muscular atrophy (SMA) can contribute to cognitive impairment.
SMA Puzzle | image credit: Dzmitry - stock.adobe.com
November 21st 2023

Innovations in SMA Treatment: The Last Decade and Beyond

Kyle Munz
The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research.
Alexander Fay, MD, PhD, UCSF Benioff Children's Hospital
November 18th 2023

Q&A: Lingering Theories and Questions in SMA Research

Kyle Munz
Alexander Fay, MD, PhD, joined for a Q&A about the current state of spinal muscular atrophy (SMA) treatment and the unanswered questions in the field.
Biomarker Concept | image credit: molekuul.be - stock.adobe.com
November 17th 2023

Researchers Identify Potential Biomarkers for SMA Motor Improvement Following Nusinersen Treatment

Kyle Munz
A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to anticipate patient outcomes.
SMA | Image credit: Rochu_2008 – stock.adobe.com
November 16th 2023

Type 1 SMA Linked to Increased Risk of Nephrolithiasis Among Children

Hayden E. Klein
According to researchers, the most prevalent risk factors in children with type 1 spinal muscular atrophy (SMA) are hypercalciuria and elevated urine specific gravity.
Infant Sitting on its Own | image credit: Prostock-studio - stock.adobe.com
October 27th 2023

RAINBOWFISH Results Highlight Risdiplam as Effective Treatment for Presymptomatic SMA

Marco Meglio
After 1 year of treatment with risdiplam, 81% of the 26-infant cohort was able to sit independently for 30 seconds, a milestone not typically reached in spinal muscular atrophy (SMA).
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