Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1 | Zendy

Pramod K. Mistry | Zendy; Elena Lukina | Zendy; Hadhami Ben Turkia | Zendy; Dominick Amato | Zendy; Hagit Baris | Zendy; Majed Dasouki | Zendy; Marwan Ghosn | Zendy; Atul Mehta | Zendy; Seymour Packman | Zendy; Gregory M. Pastores | Zendy; Мilan Petakov | Zendy; Sarit Assouline | Zendy; Manisha Balwani | Zendy; Sumita Danda | Zendy; Evgueniy Hadjiev | Zendy; Andres Ortega | Zendy; Suma P. Shankar | Zendy; María Helena Solano | Zendy; Leorah Ross | Zendy; Jennifer Angell | Zendy; Michel Peterschmitt | Zendy

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Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1

Author(s) -

Pramod K. Mistry,

Elena Lukina,

Hadhami Ben Turkia,

Dominick Amato,

Hagit Baris,

Majed Dasouki,

Marwan Ghosn,

Atul Mehta,

Seymour Packman,

Gregory M. Pastores,

Мilan Petakov,

Sarit Assouline,

Manisha Balwani,

Sumita Danda,

Evgueniy Hadjiev,

Andres Ortega,

Suma P. Shankar,

María Helena Solano,

Leorah Ross,

Jennifer Angell,

Michel Peterschmitt

Publication year - 2015

Publication title -

jama

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 4.688

H-Index - 680

eISSN - 1538-3598

pISSN - 0098-7484

DOI - 10.1001/jama.2015.459

Subject(s) - medicine , placebo , enzyme replacement therapy , anemia , hepatosplenomegaly , clinical endpoint , gastroenterology , randomized controlled trial , surgery , disease , pathology , alternative medicine

Gaucher disease type 1 is characterized by hepatosplenomegaly, anemia, thrombocytopenia, and skeletal disease. A safe, effective oral therapy is needed.

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