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Page 1
Evaluation of biomarkers for Sanfilippo syndrome.
Saville JT, Flanigan KM, Truxal KV, McBride KL, Fuller M. Saville JT, et al. Among authors: flanigan km. Mol Genet Metab. 2019 Sep-Oct;128(1-2):68-74. doi: 10.1016/j.ymgme.2019.05.005. Epub 2019 May 9. Mol Genet Metab. 2019. PMID: 31104888
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.
Murrey DA, Naughton BJ, Duncan FJ, Meadows AS, Ware TA, Campbell KJ, Bremer WG, Walker CM, Goodchild L, Bolon B, La Perle K, Flanigan KM, McBride KL, McCarty DM, Fu H. Murrey DA, et al. Among authors: flanigan km. Hum Gene Ther Clin Dev. 2014 Jun;25(2):72-84. doi: 10.1089/humc.2013.208. Epub 2014 Apr 10. Hum Gene Ther Clin Dev. 2014. PMID: 24720466 Free PMC article.
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.
Meadows AS, Duncan FJ, Camboni M, Waligura K, Montgomery C, Zaraspe K, Naughton BJ, Bremer WG, Shilling C, Walker CM, Bolon B, Flanigan KM, McBride KL, McCarty DM, Fu H. Meadows AS, et al. Among authors: flanigan km. Hum Gene Ther Clin Dev. 2015 Dec;26(4):228-42. doi: 10.1089/humc.2015.132. Hum Gene Ther Clin Dev. 2015. PMID: 26684447 Free PMC article.
A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.
Truxal KV, Fu H, McCarty DM, McNally KA, Kunkler KL, Zumberge NA, Martin L, Aylward SC, Alfano LN, Berry KM, Lowes LP, Corridore M, McKee C, McBride KL, Flanigan KM. Truxal KV, et al. Among authors: flanigan km. Mol Genet Metab. 2016 Nov;119(3):239-248. doi: 10.1016/j.ymgme.2016.08.002. Epub 2016 Aug 18. Mol Genet Metab. 2016. PMID: 27590925
Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy.
Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Fu H, et al. Among authors: flanigan km. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24. Hum Gene Ther Clin Dev. 2017. PMID: 29064732 Free PMC article.
In-frame de novo mutation in BICD2 in two patients with muscular atrophy and arthrogryposis.
Koboldt DC, Kastury RD, Waldrop MA, Kelly BJ, Mosher TM, McLaughlin H, Corsmeier D, Slaughter JL, Flanigan KM, McBride KL, Mehta L, Wilson RK, White P. Koboldt DC, et al. Among authors: flanigan km. Cold Spring Harb Mol Case Stud. 2018 Oct 1;4(5):a003160. doi: 10.1101/mcs.a003160. Print 2018 Oct. Cold Spring Harb Mol Case Stud. 2018. PMID: 30054298 Free PMC article.
Update in the Mucopolysaccharidoses.
McBride KL, Flanigan KM. McBride KL, et al. Among authors: flanigan km. Semin Pediatr Neurol. 2021 Apr;37:100874. doi: 10.1016/j.spen.2021.100874. Epub 2021 Feb 10. Semin Pediatr Neurol. 2021. PMID: 33892850 Review.
191 results