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Last Updated: 10/31/2025

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Found 976 publications

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

Reviewer Comment on Gauvreau et al. "Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen".

Reviewer Comment on Gauvreau et al. "Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen".

Journal: The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques
Published: October 03, 2025

DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice.

DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: October 01, 2025

Updates of spinal muscular atrophy in advanced therapies.

Updates of spinal muscular atrophy in advanced therapies.

Journal: Journal of the Formosan Medical Association = Taiwan yi zhi
Published: September 30, 2025

Open-label phase 4 trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy.

Open-label phase 4 trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy.

Journal: The Journal of clinical investigation
Published: September 16, 2025

State of the art: Pregnancy in spinal muscular atrophy in the treatment era.

State of the art: Pregnancy in spinal muscular atrophy in the treatment era.

Journal: Journal of neuromuscular diseases
Published: September 12, 2025

Disease-modifying therapies for spinal muscular atrophy: Family experience, ethical considerations, and the role of social determinants of health.

Disease-modifying therapies for spinal muscular atrophy: Family experience, ethical considerations, and the role of social determinants of health.

Journal: Journal of neuromuscular diseases
Published: September 03, 2025

Reply to: "A case of spinal muscular atrophy type 0 treated with nusinersen without progression of early-onset scoliosis".

Reply to: "A case of spinal muscular atrophy type 0 treated with nusinersen without progression of early-onset scoliosis".

Journal: Brain & development
Published: August 21, 2025

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database.

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database.

Journal: Children (Basel, Switzerland)
Published: July 30, 2025

Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.

Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025

Systemic Injection of Antisense Oligos into Spinal Muscular Atrophy (SMA) Mice and Evaluation.

Systemic Injection of Antisense Oligos into Spinal Muscular Atrophy (SMA) Mice and Evaluation.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025

Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.

Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Showing 1-12 of 976

Last Updated: 10/31/2025