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Megan A. Waldrop

Nationwide Children's Hospital
Ohio State University

31

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157

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Lack of toxicity in non-human primates receiving clinically relevant doses of an AAV9.U7snRNA vector designed to induce DMD exon 2 skipping.

2021 Human Gene Therapy
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Absence of significant off-target splicing variation with a U7snRNA vector targeting DMD exon 2 duplications.

2021 Human Gene Therapy
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A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

2021 medRxiv
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An evaluation of onasemnogene abeparvovec for Spinal Muscular Atrophy (SMN1)

2021 Expert opinion on orphan drugs
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Spinal muscular atrophy

2021 Seminars in Pediatric Neurology
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Validity and Reliability of the Neuromuscular Gross Motor Outcome.

2021 Pediatric Neurology
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Candidate gene modifiers of dystrophinopathy identified by the uniform application of genome-wide datasets to novel GWAS-identified loci

2021 medRxiv
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Time is muscle: A recommendation for early treatment for preterm infants with spinal muscular atrophy.

2021 Muscle & Nerve
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The genotypic and phenotypic spectrum of BICD2 variants in spinal muscular atrophy

2020 Annals of Neurology
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Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.

2020 Pediatric Pulmonology
- Nicolas J. Abreu
- Megan A. Waldrop
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Citations (9)