Issue Date | Title | Author(s) | Source | scopus | WOS | Fulltext/Archive link |
---|---|---|---|---|---|---|
2008 | Alglucosidase Alfa (Myozyme®) in Infants and Children with Rapidly Progressive Pompe Disease | Corzo D.; Byrne B.; WUH-LIANG HWU ; Leslie N.; Mandel H.; Nicolino M. | Clinical Therapeutics | 0 | 0 | |
2019 | GAA variants and phenotypes among 1,079 patients with Pompe disease: Data from the Pompe Registry | Reuser A.J.J.; van der Ploeg A.T.; YIN-HSIU CHIEN ; Llerena J.; Jr.; Abbott M.-A.; Clemens P.R.; Kimonis V.E.; Leslie N.; Maruti S.S.; Sanson B.-J.; Araujo R.; Periquet M.; Toscano A.; Kishnani P.S.; on behalf of the Pompe Registry Sites | Human Mutation | 54 | 40 | |
2007 | Recombinant human acid α-glucosidase: Major clinical benefits in infantile-onset Pompe disease | Kishnani P.S.; Corzo D.; Nicolino M.; Byrne B.; Mandel H.; WUH-LIANG HWU ; Leslie N.; Levine J.; Spencer C.; McDonald M.; Li J.; Dumontier J.; Halberthal M.; YIN-HSIU CHIEN ; Hopkin R.; Vijayaraghavan S.; Gruskin D.; Bartholomew D.; Van Der Ploeg A.; Clancy J.P.; Parini R.; Morin G.; Beck M.; De La Gastine G.S.; Jokic M.; Thurberg B.; Richards S.; Bali D.; Davison M.; Worden M.A.; Chen Y.T.; Wraith J.E. | Neurology | 661 | 583 |