Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
2017 · New England Journal of Medicine · 2,229 citations
https://doi.org/10.1056/nejmoa1706198
L
Lindsay N. Alfano
Claim or correct this author profile
Claim this profile or suggest corrections to the name, affiliation, bio, photo, or paper titles. Approved changes appear as verified CitedEvidence overlays.
213
Papers
7.6K
Citations
32
h-index
64
i10-index
Lindsay N. Alfano is an academic researcher from Nationwide Children's Hospital. The author has contributed to research in topics: Muscle Physiology and Disorders & Neurogenetic and Muscular Disorders Research & Cerebral Palsy and Movement Disorders. The author has an h-index of 32, co-authored 182 publications.
ORCID: 0000-0002-2263-7569Papers by this author
worksAVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
2019 · Journal of Neuromuscular Diseases · 176 citations
https://doi.org/10.3233/jnd-190403
272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16–18 June 2023, Hoofddorp, The Netherlands
2024 · Neuromuscular Disorders · 83 citations
https://doi.org/10.1016/j.nmd.2024.03.001
Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy
2016 · The American Journal of Human Genetics · 72 citations
https://doi.org/10.1016/j.ajhg.2016.08.023
A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design
2016 · Molecular Genetics and Metabolism · 50 citations
https://doi.org/10.1016/j.ymgme.2016.08.002
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results
2024 · Nature Medicine · 23 citations
https://doi.org/10.1038/s41591-023-02730-9
Research topics
topicsChat about Author
Papers
Chat
Click to start Chat