24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy
2013 · PLoS ONE · 130 citations
https://doi.org/10.1371/journal.pone.0052512
M
Michele Sacchini
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Michele Sacchini is an academic researcher from Meyer Children's Hospital. The author has contributed to research in topics: Lysosomal Storage Disorders Research & Glycogen Storage Diseases and Myoclonus & Neurogenetic and Muscular Disorders Research. The author has an h-index of 13, co-authored 38 publications.
Papers by this author
worksOnasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies
2023 · EClinicalMedicine · 39 citations
https://doi.org/10.1016/j.eclinm.2023.101997
Neurological assessment of newborns with spinal muscular atrophy identified through neonatal screening
2022 · European Journal of Pediatrics · 37 citations
https://doi.org/10.1007/s00431-022-04470-3
Experience of a 2-year spinal muscular atrophy NBS pilot study in Italy: towards specific guidelines and standard operating procedures for the molecular diagnosis
2022 · Journal of Medical Genetics · 36 citations
https://doi.org/10.1136/jmg-2022-108873
Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond
2023 · Orphanet Journal of Rare Diseases · 7 citations
https://doi.org/10.1186/s13023-023-02919-8
Type I spinal muscular atrophy and disease modifying treatments: a nationwide study in children born since 2016
2024 · EClinicalMedicine · 7 citations
https://doi.org/10.1016/j.eclinm.2024.102967
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