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TITLE: Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1  Full Text
AUTHORS: Pramod K Mistry; Manisha Balwani; Hagit N Baris; Hadhami Ben Turkia; Andrew A Burrow; Joel Charrow; Gerald F Cox; Sumita Danda; Marta Dragosky; Guillermo Drelichman; Amal El Beshlawy; Cristina Fraga; Selena Freisens; Sebastiaan Gaemers; Evgueniy Hadjiev; Priya S Kishnani; Elena Lukina; Pierre Maison Blanche; Ana Maria Martins; Gregory Pastores; Milan Petakov; Judith J Peterschmitt; Hanna Rosenbaum; Barry Rosenbloom; Lisa H Underhill; Timothy M Cox; ...More
PUBLISHED: 2019, SOURCE: BLOOD CELLS MOLECULES AND DISEASES, VOLUME: 77
INDEXED IN: Scopus WOS
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TITLE: Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)  Full Text
AUTHORS: Christian J Hendriksz; Roberto Giugliani; Paul Harmatz; Christina Lampe; Ana Maria Martins; Gregory M Pastores; Robert D Steiner; Elisa Leao Teles; Vassili Valayannopoulos;
PUBLISHED: 2013, SOURCE: JOURNAL OF INHERITED METABOLIC DISEASE, VOLUME: 36, ISSUE: 2
INDEXED IN: Scopus WOS CrossRef
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TITLE: Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry  Full Text
AUTHORS: William R Wilcox; Oliveira, JP ; Robert J Hopkin; Alberto Ortiz; Maryam Banikazemi; Ulla Feldt Rasmussen; Katherine Sims; Stephen Waldek; Gregory M Pastores; Philip Lee; Christine M Eng; Laszlo Marodi; Kevin E Stanford; Frank Breunig; Christoph Wanner; David G Warnock; Roberta M Lemay; Dominique P Germain;
PUBLISHED: 2008, SOURCE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 93, ISSUE: 2
INDEXED IN: Scopus WOS CrossRef: 351