Last Updated: 09/26/2026
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Found 111 publications
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
Profiling circulating Tau and phospho-Tau isoforms in pediatric and adult spinal muscular atrophy identifies selective elevation of pTau-262 in adults.
Journal: Journal of neurology
Published: March 31, 2026
Circulating Tau Profiles in Pediatric and Adult Patients with Spinal Muscular Atrophy.
Journal: medRxiv : the preprint server for health sciences
Published: January 29, 2026
Spinal muscular atrophy among US Hutterites: Phenotype variability in the setting of conserved ancestral haplotype and 4 SMN2 copies.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: June 02, 2025
Early life safety profiling of gene therapy for spinal muscular atrophy.
Journal: Gene therapy
Published: October 11, 2024
Physical performance and plasma metabolic profile as potential prognostic factors in metastatic lung cancer patients.
Journal: European journal of clinical investigation
Published: March 07, 2024
Base editing as a genetic treatment for spinal muscular atrophy.
Journal: bioRxiv : the preprint server for biology
Published: January 30, 2023
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy.
Journal: Nature biomedical engineering
Published: January 21, 2023
Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study.
Journal: Muscle & nerve
Published: August 09, 2022
264th ENMC International Workshop: Multi-system involvement in spinal muscular atrophy Hoofddorp, the Netherlands, November 19th - 21st 2021.
Journal: Neuromuscular disorders : NMD
Published: June 11, 2022
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Journal: Nature medicine
Published: February 18, 2022
Last Updated: 09/26/2026