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Frederick J. Schnell

24

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Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

2021 Nucleic Acid Therapeutics
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Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

2020 Neurology
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Challenges of Interpreting Dystrophin Content by Western Blot

2019 US neurology
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Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice

2019 Human Molecular Genetics
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Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

2019 Medicine
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Golodirsen Leads to Sarcolemmal Dystrophin Expression in Patients With Genetic Mutations Amenable to Exon 53 Skipping

2019 Pediatrics
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"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.

2018 Journal of neuromuscular diseases
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“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmdmdx/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmdmdx/J (Bl10/mdx)

2018 Journal of neuromuscular diseases
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Quantification of dystrophin to assess the effects of dystrophin-restoring treatments in Duchenne muscular dystrophy: Lessons from the development of eteplirsen

2017 European Journal of Paediatric Neurology
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Development of a validated Western blot method for quantification of human dystrophin protein used in Phase II and III clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) (P5.105)

2017 Neurology
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