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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: Cause of death in patients with attenuated acid sphingomyelinase deficiency: Comprehensive literature review and report of new cases  Full Text
AUTHORS: Jackie Imrie; Karl Eugen Mengel; David Cassiman; Paulina Mabe; Moeen Al Sayed; Bruno Bembi; Roberto Giugliani; Seymour Packman; Manuel Schiff; Tsutomu Takahashi; Hadhami Ben Turkia; Gerald F Cox;
PUBLISHED: 2016, SOURCE: 12th Annual WORLD Symposium in MOLECULAR GENETICS AND METABOLISM, VOLUME: 117, ISSUE: 2
INDEXED IN: WOS CrossRef
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TITLE: Gaucher disease in Tunisia: High frequency of the most common mutations  Full Text
AUTHORS: Wafa Cherif; Hadhami Ben Turkia; Faten Ben Rhouma; Imene Riahi; Jalel Chemli; Rym Kefi; Habib Messai; Olga Amaral; Maria Clara S Sa Miranda ; Catherine Caillaud; Neji Tebib; Marie Francoise Ben Dridi; Sonia Abdelhak;
PUBLISHED: 2009, SOURCE: BLOOD CELLS MOLECULES AND DISEASES, VOLUME: 43, ISSUE: 2
INDEXED IN: Scopus WOS CrossRef