Results 181 to 187 of about 5,278 (187)

SARA subgroup analysis based on disease duration in a phase 2 trial evaluating N-acetyl-L-leucine in GM2 gangliosidoses

Molecular Genetics and Metabolism
M. Patterson, Janelle L. Raymond, Jorgji Kerthi, Bethany Zanrucha, Asante Hatcher, T. Fields, Ian Billington, M. Strupp, T. Bremova-Ertl, K. Martakis   +9 more
semanticscholar   +1 more source

Long-term results from the phase II trial (IB1001-202) evaluating N-acetyl-L-leucine in GM2 gangliosidoses

Molecular Genetics and Metabolism
M. Patterson, Bethany Zanrucha, Janelle L. Raymond, Asante Hatcher, Jorgji Kerthi, T. Fields, Ian Billington, M. Strupp, T. Bremova-Ertl, K. Martakis   +9 more
semanticscholar   +1 more source

GM2 gangliosidoses: Developing methods for screening and monitoring the disease

Clinical Biochemistry, 2014
Denis C. Lehotay, Jennifer Billinsky, Michele Etter, Jeff Eichhorst, Jagdeep S. Walia   +4 more
openaire   +1 more source

Assay of the GM2-ganglioside cleaving hexosaminidase activity of skin fibroblasts for GM2-gangliosidoses

Clinica Chimica Acta, 1983
openaire   +2 more sources

GM2 gangliosidoses

openaire   +1 more source

New mouse models of GM2 gangliosidoses

Lab Animal
openaire   +1 more source

Targeting the hexosaminidase genes: mouse models of the GM2 gangliosidoses.

Advances in genetics, 2002
openaire   +1 more source