Megan A. Waldrop

Neuromusculoskeletal Medicine

555 S 18th St
Columbus, OH 43205

Last Updated: 09/26/2026

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Found 46 publications

Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.

Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.

Journal: Journal of comparative effectiveness research

Published: April 10, 2026

Characterizing Friedreich's ataxia cardiomyopathy with serial cardiac magnetic resonance imagings.

Characterizing Friedreich's ataxia cardiomyopathy with serial cardiac magnetic resonance imagings.

Journal: Annals of pediatric cardiology

Published: January 10, 2026

Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry.

Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry.

Journal: Journal of neuromuscular diseases

Published: November 11, 2025

Gene x environment interaction analysis confirms genetic modifier effects on steroid efficacy via TGF-β pathway in Duchenne muscular dystrophy.

Gene x environment interaction analysis confirms genetic modifier effects on steroid efficacy via TGF-β pathway in Duchenne muscular dystrophy.

Journal: European journal of human genetics : EJHG

Published: September 23, 2025

Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study.

Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study.

Journal: EClinicalMedicine

Published: July 31, 2025

Gross motor delays in infants and young boys with Duchenne muscular dystrophy.

Gross motor delays in infants and young boys with Duchenne muscular dystrophy.

Journal: Journal of neuromuscular diseases

Published: February 20, 2025

Current clinical applications of AAV-mediated gene therapy.

Current clinical applications of AAV-mediated gene therapy.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: January 17, 2025

Heterozygous loss-of-function variants in SPTAN1 cause an early childhood onset distal myopathy.

Heterozygous loss-of-function variants in SPTAN1 cause an early childhood onset distal myopathy.

Journal: Genetics in medicine : official journal of the American College of Medical Genetics

Published: October 15, 2024

Heterozygous loss-of-function variants in SPTAN1 cause a novel early childhood onset distal myopathy with chronic neurogenic features.

Heterozygous loss-of-function variants in SPTAN1 cause a novel early childhood onset distal myopathy with chronic neurogenic features.

Journal: medRxiv : the preprint server for health sciences

Published: October 07, 2024

Comparison of Two Questionnaires for Sleep-Related Symptoms in Pediatric and Adult Patients With Myotonic Dystrophy Type 1.

Comparison of Two Questionnaires for Sleep-Related Symptoms in Pediatric and Adult Patients With Myotonic Dystrophy Type 1.

Journal: Muscle & nerve

Published: April 29, 2024

Clinical decision making around commercial use of gene and genetic therapies for spinal muscular atrophy.

Clinical decision making around commercial use of gene and genetic therapies for spinal muscular atrophy.

Journal: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Published: April 01, 2024

Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study.

Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study.

Journal: Journal of neuromuscular diseases

Published: March 10, 2024

Showing 1-12 of 46

Last Updated: 09/26/2026