Title: | Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease |
Journal : | Genetics in medicine : official journal of the American College of Medical Genetics |
Authors: | Kishnani PS, Author ; Gibson JB ; Gambello MJ ; Hillman R ; Stockton DW ; Kronn D ; Leslie ND ; Pena LDM ; Tanpaiboon P ; Day JW ; Wang RY ; Goldstein JL ; An Haack K ; Sparks SE ; Zhao Y ; Hahn SH |
Material Type: | Article |
Publication Date: | 14/05/2019 |
Languages: | English |
Pubmed / DOI : | DOI : 10.1038/s41436-019-0527-9 / Pubmed : 31086307 |
N° Profil MNM : | 2019052 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31086307 |