Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy

Cerebral Mitochondrial Microangiopathy Leads to Leukoencephalopathy in Mitochondrial Neurogastrointestinal Encephalopathy

(2018) L.L. Gramegna et al.   AMERICAN JOURNAL OF NEURORADIOLOGY

Challenges related to the immunogenicity of parenteral recombinant proteins: Underlying mechanisms and new approaches to overcome it

(2018) Fatemeh Faraji et al.   INTERNATIONAL REVIEWS OF IMMUNOLOGY

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

(2018) Rana Yadak et al.   Molecular Therapy-Methods & Clinical Development

Validation of an immunoassay for anti-thymidine phosphorylase antibodies in patients with MNGIE treated with enzyme replacement therapy

(2018) Michelle Levene et al.   Molecular Therapy-Methods & Clinical Development

Mitochondrial Neurogastrointestinal Encephalomyopathy: Into the Fourth Decade, What We Have Learned So Far

(2018) Dario Pacitti et al.   Frontiers in Genetics

Liver transplantation for mitochondrial neurogastrointestinal encephalomyopathy

(2016) Roberto De Giorgio et al.   ANNALS OF NEUROLOGY

Long-Term Restoration of Thymidine Phosphorylase Function and Nucleoside Homeostasis Using Hematopoietic Gene Therapy in a Murine Model of Mitochondrial Neurogastrointestinal Encephalomyopathy

(2016) Javier Torres-Torronteras et al.   HUMAN GENE THERAPY

Allogeneic haematopoietic stem cell transplantation for mitochondrial neurogastrointestinal encephalomyopathy

(2015) Joerg P. Halter et al.   BRAIN

Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement

(2013) B. E. Bax et al.   NEUROLOGY

Course and management of allogeneic stem cell transplantation in patients with mitochondrial neurogastrointestinal encephalomyopathy

(2012) Massimiliano Filosto et al.   JOURNAL OF NEUROLOGY

Validation of a HPLC method for the measurement of erythrocyte encapsulated thymidine phosphorylase (EE-TP) activity

(2012) Lynette D. Fairbanks et al.   JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Clinical and genetic spectrum of mitochondrial neurogastrointestinal encephalomyopathy

(2011) Caterina Garone et al.   BRAIN

International seminar on the red blood cells as vehicles for drugs

(2011) Yann Godfrin et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach

(2010) J Halter et al.   BONE MARROW TRANSPLANTATION

CARRIER ERYTHROCYTE ENTRAPPED THYMIDINE PHOSPHORYLASE THERAPY FOR MNGIE

(2008) N. F. Moran et al.   NEUROLOGY