Spinal Muscular Atrophy Type 1 Latest Advances

Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility.

Journal: Neuromuscular disorders : NMD

Published: May 09, 2026

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

Journal: Developmental medicine and child neurology

Published: April 09, 2026

Ceftriaxone-Resistant Escherichia coli Sepsis in an Infant With Spinal Muscular Atrophy Type 1 Receiving Risdiplam: A Case Report.

Journal: Clinical case reports

Published: March 12, 2026

Safety and Efficacy of Magnetically Controlled Growing Rods for Early Onset Scoliosis in the Era of Disease-Modifying Therapies: Mid-term Results in a Homogeneous Cohort of Spinal Muscular Atrophy Type 1 Patients.

Journal: Journal of the Pediatric Orthopaedic Society of North America

Published: March 08, 2026

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

Journal: Developmental medicine and child neurology

Published: February 01, 2026

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Journal: The Lancet. Child & adolescent health

Published: January 28, 2026

Swallowing assessment in spinal muscular atrophy type 1: a real-world study of dysphagia in children receiving disease-modifying therapies.

Journal: Neuromuscular disorders : NMD

Published: January 27, 2026

Fusionless spinal surgery in children with spinal muscular atrophy type 1 with bipolar system: a preliminary communication.

Journal: Journal of pediatric orthopedics. Part B

Published: January 22, 2026

Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy.

Journal: Pediatric pulmonology

Published: December 22, 2025

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.

Journal: Neuromuscular disorders : NMD

Published: October 21, 2025

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

Journal: JAMA network open

Published: October 08, 2025

Spinal Muscular Atrophy Type 1 Latest Advances

Find the Latest Research About Spinal Muscular Atrophy Type 1

Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility.

Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility.

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.

Ceftriaxone-Resistant Escherichia coli Sepsis in an Infant With Spinal Muscular Atrophy Type 1 Receiving Risdiplam: A Case Report.

Ceftriaxone-Resistant Escherichia coli Sepsis in an Infant With Spinal Muscular Atrophy Type 1 Receiving Risdiplam: A Case Report.

Safety and Efficacy of Magnetically Controlled Growing Rods for Early Onset Scoliosis in the Era of Disease-Modifying Therapies: Mid-term Results in a Homogeneous Cohort of Spinal Muscular Atrophy Type 1 Patients.

Safety and Efficacy of Magnetically Controlled Growing Rods for Early Onset Scoliosis in the Era of Disease-Modifying Therapies: Mid-term Results in a Homogeneous Cohort of Spinal Muscular Atrophy Type 1 Patients.

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

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

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Swallowing assessment in spinal muscular atrophy type 1: a real-world study of dysphagia in children receiving disease-modifying therapies.

Swallowing assessment in spinal muscular atrophy type 1: a real-world study of dysphagia in children receiving disease-modifying therapies.

Fusionless spinal surgery in children with spinal muscular atrophy type 1 with bipolar system: a preliminary communication.

Fusionless spinal surgery in children with spinal muscular atrophy type 1 with bipolar system: a preliminary communication.

Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy.

Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy.

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.

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.

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.