E. Wraith

154. Disease stability in patients with Niemann–Pick disease type C treated with miglustat

M. Patterson, E. Wraith, D. Vecchio, 2009 .

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.

A. Fischer, J. Tolar, J. Kurtzberg, 2013, Blood.

Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease

E. Wraith, K. Tylee, R. Wynn, 2007, Bone Marrow Transplantation.

Regional anesthetic techniques are an alternative to general anesthesia for infants with Pompe's disease

E. Wraith, J. Bruce, R. Walker, 2007, Paediatric anaesthesia.

Kinematic analysis of motor dysfunction in Niemann-Pick type C

M. Patterson, S. Pullman, Q. Yu, 2007, Clinical Neurophysiology.

111. Miglustat in Niemann-Pick disease type C (NPC)

M. Patterson, L. Abel, D. Vecchio, 2008 .

Initial Human Experience with SBC-102, a Recombinant Enzyme Replacement Therapy in Adults with Lysosomal Acid Lipase Deficiency

G. Enns, M. Balwani, E. Schneider, 2012 .