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Spinal Muscular Atrophy.
Nicolau S, Waldrop MA, Connolly AM, Mendell JR. Nicolau S, et al. Among authors: mendell jr. Semin Pediatr Neurol. 2021 Apr;37:100878. doi: 10.1016/j.spen.2021.100878. Epub 2021 Feb 11. Semin Pediatr Neurol. 2021. PMID: 33892848 Review.
One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development.
Connolly AM, Florence JM, Cradock MM, Eagle M, Flanigan KM, McDonald CM, Karachunski PI, Darras BT, Bushby K, Malkus EC, Golumbek PT, Zaidman CM, Miller JP, Mendell JR; MDA DMD Clinical Research Network. Connolly AM, et al. Among authors: mendell jr. Pediatr Neurol. 2014 Jun;50(6):557-63. doi: 10.1016/j.pediatrneurol.2014.02.006. Epub 2014 Feb 15. Pediatr Neurol. 2014. PMID: 24842254 Free PMC article.
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.
Waldrop MA, Karingada C, Storey MA, Powers B, Iammarino MA, Miller NF, Alfano LN, Noritz G, Rossman I, Ginsberg M, Mosher KA, Broomall E, Goldstein J, Bass N, Lowes LP, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Among authors: mendell jr. Pediatrics. 2020 Sep;146(3):e20200729. doi: 10.1542/peds.2020-0729. Pediatrics. 2020. PMID: 32843442
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Peña LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Day JW, et al. Among authors: mendell jr. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17. Lancet Neurol. 2021. PMID: 33743238 Clinical Trial.
Therapeutic Approaches for Duchenne Muscular Dystrophy: Old and New.
Mackenzie SJ, Nicolau S, Connolly AM, Mendell JR. Mackenzie SJ, et al. Among authors: mendell jr. Semin Pediatr Neurol. 2021 Apr;37:100877. doi: 10.1016/j.spen.2021.100877. Epub 2021 Feb 11. Semin Pediatr Neurol. 2021. PMID: 33892842 Review.
Validity and Reliability of the Neuromuscular Gross Motor Outcome.
Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Alfano LN, et al. Among authors: mendell jr. Pediatr Neurol. 2021 Sep;122:21-26. doi: 10.1016/j.pediatrneurol.2021.05.021. Epub 2021 Jun 8. Pediatr Neurol. 2021. PMID: 34271497
Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene.
Flanigan KM, Dunn DM, von Niederhausern A, Soltanzadeh P, Howard MT, Sampson JB, Swoboda KJ, Bromberg MB, Mendell JR, Taylor LE, Anderson CB, Pestronk A, Florence JM, Connolly AM, Mathews KD, Wong B, Finkel RS, Bonnemann CG, Day JW, McDonald C; United Dystrophinopathy Project Consortium; Weiss RB. Flanigan KM, et al. Among authors: mendell jr. Hum Mutat. 2011 Mar;32(3):299-308. doi: 10.1002/humu.21426. Hum Mutat. 2011. PMID: 21972111 Free PMC article.
LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophy.
Flanigan KM, Ceco E, Lamar KM, Kaminoh Y, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Gappmaier E, Howard MT, Day JW, McDonald C, McNally EM, Weiss RB; United Dystrophinopathy Project. Flanigan KM, et al. Among authors: mendell jr. Ann Neurol. 2013 Apr;73(4):481-8. doi: 10.1002/ana.23819. Epub 2013 Feb 20. Ann Neurol. 2013. PMID: 23440719 Free PMC article.
408 results