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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14.
Lancet. 2017.
PMID: 28712537
Free PMC article.
Clinical Trial.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA.
George LA, et al. Among authors: wachtel k.
N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.
N Engl J Med. 2017.
PMID: 29211678
Free PMC article.
Clinical Trial.
Item in Clipboard
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BJ, Ozelo M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA.
George LA, et al. Among authors: wachtel k.
Mol Ther. 2020 Sep 2;28(9):2073-2082. doi: 10.1016/j.ymthe.2020.06.001. Epub 2020 Jun 10.
Mol Ther. 2020.
PMID: 32559433
Free PMC article.
Clinical Trial.
Item in Clipboard
Reaction to diagnosis and parenting styles among mothers of young children with ASDs.
Wachtel K, Carter AS.
Wachtel K, et al.
Autism. 2008 Sep;12(5):575-94. doi: 10.1177/1362361308094505.
Autism. 2008.
PMID: 18805948
Item in Clipboard
Inability to alkalinize urine in a patient at risk for tumor lysis syndrome: a case report.
Steelman R, Pate MF, Shoun P, Wachtel K, Johannes PW.
Steelman R, et al. Among authors: wachtel k.
Pediatr Hematol Oncol. 2008 Mar;25(2):155-8. doi: 10.1080/08880010701884774.
Pediatr Hematol Oncol. 2008.
PMID: 18363183
Item in Clipboard
A rapid cation-exchange HPLC method for detection and quantification of pyridinium oximes in plasma and tissue.
Singh H, Moorad-Doctor D, Ratcliffe RH, Wachtel K, Castillo A, Garcia GE.
Singh H, et al. Among authors: wachtel k.
J Anal Toxicol. 2007 Mar;31(2):69-74. doi: 10.1093/jat/31.2.69.
J Anal Toxicol. 2007.
PMID: 17536740
Item in Clipboard
The Brief Infant-Toddler Social and Emotional Assessment: screening for social-emotional problems and delays in competence.
Briggs-Gowan MJ, Carter AS, Irwin JR, Wachtel K, Cicchetti DV.
Briggs-Gowan MJ, et al. Among authors: wachtel k.
J Pediatr Psychol. 2004 Mar;29(2):143-55. doi: 10.1093/jpepsy/jsh017.
J Pediatr Psychol. 2004.
PMID: 15096535
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