Spinal Muscular Atrophy Type 1 Latest Advances

Find the Latest Research About Spinal Muscular Atrophy Type 1

Last Updated: 04/28/2026

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Found 290 publications

Challenges in assessing autism spectrum disorder in spinal muscular atrophy type 1.

Challenges in assessing autism spectrum disorder in spinal muscular atrophy type 1.

Journal: Developmental medicine and child neurology
Published: February 01, 2026

285th ENMC international workshop: SMN-associated neurodevelopmental disorder: type 1 spinal muscular atrophy and the brain, 31st January - 2nd February 2025, Hoofddorp, The Netherlands.

285th ENMC international workshop: SMN-associated neurodevelopmental disorder: type 1 spinal muscular atrophy and the brain, 31st January - 2nd February 2025, Hoofddorp, The Netherlands.

Journal: Neuromuscular disorders : NMD
Published: December 02, 2025

Longitudinal assessment of respiratory status utilising the amended Great Ormond Street Respiratory Score in treated spinal muscular atrophy type 1 children.

Longitudinal assessment of respiratory status utilising the amended Great Ormond Street Respiratory Score in treated spinal muscular atrophy type 1 children.

Journal: Neuromuscular disorders : NMD
Published: October 21, 2025

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

Journal: JAMA network open
Published: October 08, 2025

Elexacaftor/Tezacaftor/Ivacaftor Therapy in a Child With Cystic Fibrosis and Type 1 Spinal Muscular Atrophy.

Elexacaftor/Tezacaftor/Ivacaftor Therapy in a Child With Cystic Fibrosis and Type 1 Spinal Muscular Atrophy.

Journal: Pediatric pulmonology
Published: October 07, 2025

Autism spectrum disorder in children with spinal muscular atrophy type 1: Case series.

Autism spectrum disorder in children with spinal muscular atrophy type 1: Case series.

Journal: Developmental medicine and child neurology
Published: September 25, 2025

Adult Survival in SMA Type 1: A 23-Year Journey With Home Ventilation and Multidisciplinary Support.

Adult Survival in SMA Type 1: A 23-Year Journey With Home Ventilation and Multidisciplinary Support.

Journal: Clinical case reports
Published: August 28, 2025

Comparison of Nusinersen Monotherapy Versus Combination Therapy With Nusinersen and Onasemnogene Abeparvovec in Spinal Muscular Atrophy Type 1 Patients With Two SMN2 Copies: A Multicenter Study From Türkiye.

Comparison of Nusinersen Monotherapy Versus Combination Therapy With Nusinersen and Onasemnogene Abeparvovec in Spinal Muscular Atrophy Type 1 Patients With Two SMN2 Copies: A Multicenter Study From Türkiye.

Journal: Muscle & nerve
Published: July 07, 2025

Early Intervention and Speed-to-Effect in Spinal Muscular Atrophy Type 1 Following Onasemnogene Abeparvovec Gene Replacement Therapy: Results of aPost-Hoc Analysis of Pooled Clinical Study Data.

Early Intervention and Speed-to-Effect in Spinal Muscular Atrophy Type 1 Following Onasemnogene Abeparvovec Gene Replacement Therapy: Results of aPost-Hoc Analysis of Pooled Clinical Study Data.

Journal: Neurology and therapy
Published: May 14, 2025

Nusinersen rescues taurine deficiency in patients with type 1 Spinal Muscular Atrophy.

Nusinersen rescues taurine deficiency in patients with type 1 Spinal Muscular Atrophy.

Journal: Communications medicine
Published: April 30, 2025

Bereavement Experiences Among Parents of Children With Spinal Muscular Atrophy Type 1.

Bereavement Experiences Among Parents of Children With Spinal Muscular Atrophy Type 1.

Journal: Journal of pain and symptom management
Published: April 11, 2025

The effect of thoracolumbosacral orthosis on scoliosis progression and chest deformity in children with type 1 spinal muscular atrophy: A randomized controlled trial.

The effect of thoracolumbosacral orthosis on scoliosis progression and chest deformity in children with type 1 spinal muscular atrophy: A randomized controlled trial.

Journal: PloS one
Published: April 02, 2025
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Last Updated: 04/28/2026