N. Miller

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman et al. · JAMA Neurology · 2020 · 392 citations · Full text

Pharmacy, Pediatrics, Pharmacotherapy +17

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

Megan A. Waldrop, Cassandra Karingada, Mike A. Storey et al. · PEDIATRICS · 2020 · 134 citations

Medicine, Genetics, Pediatrics +16

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes

Jerry R. Mendell, Zarife Sahenk, Samiah Al-Zaidy et al. · Molecular Therapy · 2017 · 105 citations · Full text

Medicine, Orthopedic Surgery, Rehabilitation Medicine +17

Clinical outcomes and response of patients applying topical therapy for pyoderma gangrenosum: A prospective cohort study

Kim S Thomas, Anthony D. Ormerod, Fiona E. Craig et al. · Journal of the American Academy of Dermatology · 2016 · 64 citations · Full text

Medicine, Clinical Sciences, Dermatology +10

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion

Jerry R. Mendell, Louis G. Chicoine, Samiah Al-Zaidy et al. · Human Gene Therapy · 2019 · 45 citations · Full text

Life Sciences, Biotechnology, Genetics +17