Last Updated: 09/26/2026
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Found 20 publications
Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age Group-Matched Controls.
Journal: Journal of child neurology
Published: October 09, 2020
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: August 12, 2020
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Journal: JAMA neurology
Published: June 16, 2020
Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing.
Journal: International journal of cardiology
Published: January 31, 2020
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
Journal: Journal of neuromuscular diseases
Published: August 06, 2019
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.
Journal: Pediatric neurology
Published: March 22, 2019
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
Journal: Human gene therapy
Published: March 07, 2019
From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1.
Journal: Pediatric neurology
Published: February 06, 2019
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy.
Journal: Pediatric pulmonology
Published: June 22, 2018
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Journal: The New England journal of medicine
Published: November 02, 2017
A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy.
Journal: International journal of neonatal screening
Published: June 01, 2017
Last Updated: 09/26/2026