Fatbardha Varfaj

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

Perry B. Shieh, Nancy L. Kuntz, Wolfgang Müller‐Felber et al. · The Lancet Neurology · 2023 · 120 citations · Full text

Medicine, Molecular Medicine, Pharmacotherapy +15

Generation of enhanced stability factor VIII variants by replacement of charged residues at the A2 domain interface

Hironao Wakabayashi, Fatbardha Varfaj, Jennifer P. DeAngelis et al. · Blood · 2008 · 45 citations · Full text

Life Sciences, Genetics, Molecular Biology +11

Carbon-Carbon Bond Cleavage in Activation of the Prodrug Nabumetone

Fatbardha Varfaj, Siti N. A. Zulkifli, Hyoung‐Goo Park et al. · Drug Metabolism and Disposition · 2014 · 43 citations

Life Sciences, Biochemistry, Pharmacology +17

Role of P1 residues Arg336 and Arg562 in the activated-Protein-C-catalysed inactivation of Factor VIIIa

Fatbardha Varfaj, Julie Neuberg, P. Vincent Jenkins et al. · Biochemical Journal · 2006 · 30 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +13

Role of Cysteine Residues in Heme Binding to Human Heme Oxygenase-2 Elucidated by Two-dimensional NMR Spectroscopy

Fatbardha Varfaj, Jed N. Lampe, Paul R. Ortiz de Montellano · Journal of Biological Chemistry · 2012 · 29 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +11