Spinal Muscular Atrophy (SMA) Latest Advances

Out-of-Pocket Cost of Spinal Muscular Atrophy Gene Therapies Among Commercially Insured Patients.

Journal: JAMA pediatrics

Published: April 13, 2026

Navigating new motor function trajectories: Consensus recommendations for assessment in the era of newborn screening and early treatment in SMA.

Journal: Journal of neuromuscular diseases

Published: April 07, 2026

Incidence, Timing and Clinical Significance of Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis.

Journal: Circulation. Heart failure

Published: April 07, 2026

Spatial transcriptomics identifies dysregulated programs across neural and non-neural tissues in spinal muscular atrophy.

Journal: bioRxiv : the preprint server for biology

Published: April 03, 2026

Longitudinal Analysis of Superoxide Dismutase 1 Seeding Activity in Amyotrophic Lateral Sclerosis Cerebrospinal Fluid.

Journal: medRxiv : the preprint server for health sciences

Published: April 03, 2026

Astrocyte targeted SMN1 gene therapy and forskolin application improves astrocyte filopodia actin defects and motor neuron synaptic dysfunction in human SMA disease pathology.

Journal: bioRxiv : the preprint server for biology

Published: April 03, 2026

Cardiac defects in spinal muscular atrophy and the role of SMN in cardiomyocyte homeostasis.

Journal: bioRxiv : the preprint server for biology

Published: April 03, 2026

Correction: Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam.

Journal: Journal of neurology

Published: March 28, 2026

Corrigendum to "Learnings from a registry-based cohort study for spinal muscular atrophy disease" [Neuromuscular Disorders, Volume 59, February 2026, 106332].

Journal: Neuromuscular disorders : NMD

Published: March 28, 2026

KIF5A downregulation in spinal muscular atrophy links axonal regeneration defects with ALS.

Journal: JCI insight

Published: March 26, 2026

Upper and Middle Thoracic Esophageal Dilation in patients with Spinal and Bulbar Muscular Atrophy.

Journal: Internal medicine (Tokyo, Japan)

Published: March 26, 2026

Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic.

Journal: Proceedings of the National Academy of Sciences of the United States of America

Published: March 26, 2026

Spinal Muscular Atrophy (SMA) Latest Advances

Find the Latest Research About Spinal Muscular Atrophy (SMA)

Out-of-Pocket Cost of Spinal Muscular Atrophy Gene Therapies Among Commercially Insured Patients.

Out-of-Pocket Cost of Spinal Muscular Atrophy Gene Therapies Among Commercially Insured Patients.

Navigating new motor function trajectories: Consensus recommendations for assessment in the era of newborn screening and early treatment in SMA.

Navigating new motor function trajectories: Consensus recommendations for assessment in the era of newborn screening and early treatment in SMA.

Incidence, Timing and Clinical Significance of Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis.

Incidence, Timing and Clinical Significance of Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis.

Spatial transcriptomics identifies dysregulated programs across neural and non-neural tissues in spinal muscular atrophy.

Spatial transcriptomics identifies dysregulated programs across neural and non-neural tissues in spinal muscular atrophy.

Longitudinal Analysis of Superoxide Dismutase 1 Seeding Activity in Amyotrophic Lateral Sclerosis Cerebrospinal Fluid.

Longitudinal Analysis of Superoxide Dismutase 1 Seeding Activity in Amyotrophic Lateral Sclerosis Cerebrospinal Fluid.

Astrocyte targeted SMN1 gene therapy and forskolin application improves astrocyte filopodia actin defects and motor neuron synaptic dysfunction in human SMA disease pathology.

Astrocyte targeted SMN1 gene therapy and forskolin application improves astrocyte filopodia actin defects and motor neuron synaptic dysfunction in human SMA disease pathology.

Cardiac defects in spinal muscular atrophy and the role of SMN in cardiomyocyte homeostasis.

Cardiac defects in spinal muscular atrophy and the role of SMN in cardiomyocyte homeostasis.

Correction: Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam.

Correction: Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam.

Corrigendum to "Learnings from a registry-based cohort study for spinal muscular atrophy disease" [Neuromuscular Disorders, Volume 59, February 2026, 106332].

Corrigendum to "Learnings from a registry-based cohort study for spinal muscular atrophy disease" [Neuromuscular Disorders, Volume 59, February 2026, 106332].

KIF5A downregulation in spinal muscular atrophy links axonal regeneration defects with ALS.

KIF5A downregulation in spinal muscular atrophy links axonal regeneration defects with ALS.

Upper and Middle Thoracic Esophageal Dilation in patients with Spinal and Bulbar Muscular Atrophy.

Upper and Middle Thoracic Esophageal Dilation in patients with Spinal and Bulbar Muscular Atrophy.

Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic.

Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic.