Results 181 to 187 of about 8,071 (187)

Chondrogenesis and osteogenesis are delayed by MPS IVA keratan sulphate but not normal keratan sulphate

Molecular Genetics and Metabolism, 2018
Sharon Byers, Srimayee Vaidyanathan, Ainslie L.K. Derrick-Roberts   +2 more
openaire   +1 more source

The performance characteristics of a 6-plex assay for the detection of six lysosomal diseases and preliminary data for the detection of MPS II, MPS IVA and MPS VI

Molecular Genetics and Metabolism, 2016
C. Ronald Scott, Susan Elliott, Zdenek Spacil, Sophia Masai, Yang Liu, Hsuan-Chieh Liao, Arun Babu Kumar, Naveen Chennamaneni, Norman Buroker, Jie-Yu Huang, Jie Feng, Aaron Boyce, Jason Cournoyer, Frantisek Turecek, Michael H. Gelb   +14 more
openaire   +1 more source

The patient experience of elosulfase alfa treatment for MPS IVA (Morquio syndrome type A): beyond traditional measures

Molecular Genetics and Metabolism, 2017
James Jarrett, Mohit Jain, Charlotte Roberts, Alexandra Morrison   +3 more
openaire   +1 more source

All-in-one lentiviral vectors improving bone abnormalities in MPS IVA mice

Molecular Genetics and Metabolism
Betul Celik, Sampurna Saikia, Fnu Nidhi, Shaukat Khan, Shunji Tomatsu   +4 more
openaire   +1 more source

In vivo direct bone targeting lentiviral gene therapy for MPS IVA murine model

Molecular Genetics and Metabolism
Betul Celik, Sampurna Saikia, Fnu Nidhi, Shaukat Khan, Shunji Tomatsu   +4 more
openaire   +1 more source

Frequent GAG monitoring to evaluate the efficacy of enzyme replacement therapy in MPS IVA

Molecular Genetics and Metabolism
Nishitha R. Pillai, Jeanine R. Jarnes, Alia Ahmed, Chester B. Whitley   +3 more
openaire   +1 more source

Phenotypic findings related to the c.1139 + 1 G > A variant in four individuals with MPS IVA

Molecular Genetics and Metabolism
openaire   +1 more source