Liesl Mathias

Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

Christian Braun, Kaan Boztuǧ, Anna Paruzynski et al. · Science Translational Medicine · 2014 · 555 citations · Full text

Medicine, Genetics, Molecular Genetics +17

Long‐term safety and efficacy of deferasirox (Exjade^®) for up to 5 years in transfusional iron‐overloaded patients with sickle cell disease

Elliott Vichinsky, Françoise Bernaudin, Renée V. Gardner et al. · British Journal of Haematology · 2011 · 78 citations · Full text

Medicine, Hematology, Pharmacology +17

Treatment of recurrent clear cell sarcoma of the kidney with brain metastasis

Vlad C. Radulescu, Mary Gerrard, Chris Moertel et al. · Pediatric Blood & Cancer · 2007 · 49 citations

Medicine, Oncology, Surgery +17

Laboratory variability in the diagnosis of type 2 VWD variants

Stefanie DiGiandomenico, Pamela A. Christopherson, Sandra L. Haberichter et al. · Journal of Thrombosis and Haemostasis · 2020 · 21 citations · Full text

Life Sciences, Genetics, Human Genetics +10

Molecular pathogenesis and heterogeneity in type 3 VWD families in U.S. Zimmerman program

Pamela A. Christopherson, Sandra L. Haberichter, Veronica H. Flood et al. · Journal of Thrombosis and Haemostasis · 2022 · 21 citations · Full text

Life Sciences, Genetics, Molecular Genetics +7