Page 9 - Read Online
P. 9

Angelini. J Transl Genet Genom 2024;8:244-8  https://dx.doi.org/10.20517/jtgg.2024.29  Page 248

               Copyright
               © The Author(s) 2024.


               REFERENCES
               1.       Bonifati DM, Witchel SF, Ermani M, Hoffman EP, Angelini C, Pegoraro E. The glucocorticoid receptor N363S polymorphism and
                   steroid response in Duchenne dystrophy. J Neurol Neurosurg Psychiatry 2006;77:1177-9.  DOI  PubMed
               2.       Biggar WD, Gingras M, Fehlings DL, Harris VA, Steele CA. Deflazacort treatment of Duchenne muscular dystrophy. J Pediatr
                   2001;138:45-50.  DOI  PubMed
               3.       Guglieri M, Bushby K, McDermott MP, et al. Effect of different corticosteroid dosing regimens on clinical outcomes in boys with
                   duchenne muscular dystrophy: a randomized clinical trial. JAMA 2022;327:1456-68.  DOI
               4.       Smith EC, Conklin LS, Hoffman EP, et al. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: an 18-month interim
                   analysis of a non-randomized open-label extension study. PLoS Med 2020;17:e1003222.  DOI  PubMed  PMC
               5.       Angelini  C,  Pinzan  E.  Advances  in  imaging  of  brain  abnormalities  in  neuromuscular  disease.  Ther  Adv  Neurol  Disord
                   2019;12:1756286419845567.  DOI  PubMed  PMC
               6.       Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY. Corticosteroids for the treatment of Duchenne muscular dystrophy.
                   Cochrane Database Syst Rev 2016;2016:CD003725.  DOI  PubMed  PMC
               7.       McDonald CM, Henricson EK, Abresch RT, et al. Long-term effects of glucocorticoids on function, quality of life, and survival in
                   patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet 2018;391:451-61.  DOI
               8.       Okubo M, Noguchi S, Hayashi S, et al. Exon skipping induced by nonsense/frameshift mutations in DMD gene results in Becker
                   muscular dystrophy. Hum Genet 2020;139:247-55.  DOI  PubMed  PMC
               9.       Sheikh O, Yokota T. Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update. Arch Toxicol
                   2022;96:1-9.  DOI  PubMed
               10.      Straub V, Guglieri M. An update on Becker muscular dystrophy. Curr Opin Neurol 2023;36:450-4.  DOI  PubMed  PMC
   4   5   6   7   8   9   10   11   12   13   14