Filters
Term Search
You can select from the dropdown list OR enter your own terms to refine the search.
Last Updated: 10/31/2025
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 281 publications
Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.
Journal: JAMA network open
Published: October 08, 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.
Journal: Neurology and therapy
Published: May 14, 2025
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.
Journal: PloS one
Published: April 02, 2025
The clinical and genetic profiles of spinal muscular atrophy with respiratory distress type 1 and identification of a novel mutation in IGHMBP2 in China.
Journal: Frontiers in genetics
Published: March 15, 2025
Safety of Onasemnogene Abeparvovec Administration to Type 1 SMA Patients Who Have Received Risdiplam.
Journal: Journal of clinical neuromuscular disease
Published: February 26, 2025
Families' Perceptions of Powered Mobility for Participation in Children With Spinal Muscular Atrophy Type 1: A Photovoice Study.
Journal: Health expectations : an international journal of public participation in health care and health policy
Published: February 21, 2025
Eosinophilic Esophagitis in a 3-Year-Old Girl with Spinal Muscular Atrophy Type 1: The First Reported Case.
Journal: Pediatric reports
Published: February 16, 2025
Spinal presentations in children with type 1 spinal muscular atrophy on nusinersen treatment across the SMA-REACH UK network: a retrospective national observational study.
Journal: BMJ open
Published: January 22, 2025
Expressive language and social communication abilities in children with spinal muscular atrophy type 1.
Journal: Developmental medicine and child neurology
Published: December 23, 2024
The long-term efficacy of nusinersen on respiratory functions in children with symptomatic spinal muscular atrophy type 1.
Journal: Turkish journal of medical sciences
Published: November 22, 2024
Could choosing risdiplam instead of nusinersen in the treatment of type 1 spinal muscular atrophy be a huge cost-minimization opportunity?
Journal: Croatian medical journal
Published: November 04, 2024
Last Updated: 10/31/2025