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

Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia, +18

JAMA

Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with <scp>G</scp>aucher disease type 1: The phase 3 ENGAGE trial

Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia, +15

American Journal of Hematology

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study

Derlis Gonzalez, Hadhami Ben Turkia, Elena Lukina, +8

American Journal of Hematology

Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease

Hadhami Ben Turkia, Derlis Gonzalez, Norman W. Barton, +14

American Journal of Hematology

ImmunohematologyGaucher DiseaseEnzyme Replacement TherapyTransfusion MedicineHematology +6

Clinical outcomes after 4.5 years of eliglustat therapy for <scp>Gaucher</scp> disease type 1: Phase 3 <scp>ENGAGE</scp> trial final results

Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia, +15

American Journal of Hematology

Treatment-naïve Gaucher disease patients achieve therapeutic goals and normalization with velaglucerase alfa by 4 years in phase 3 trials

Ari Zimran, Deborah Elstein, Derlis Gonzalez, +4

Blood Cells Molecules and Diseases

Enzyme Replacement TherapySubstrate Reduction TherapyOsteoporosisOrthopaedic SurgeryType 1 +14

Molecular characterization of MPS IIIA, MPS IIIB and MPS IIIC in Tunisian patients

Souad Ouesleti, Valéry Brunel, Hadhami Ben Turkia, +7

Clinica Chimica Acta

Mps IiicMps IiiaMedicinePathologyMps Iiib +4

Phenotypic spectrum of fucosidosis in Tunisia

Hadhami Ben Turkia, Néji Tebib, Hatem Azzouz, +8

Journal of Inherited Metabolic Disease

Mucopolysaccharidosis type I: molecular characteristics of two novel alpha-L-iduronidase mutations in Tunisian patients

Latifa Chkioua, Souhir Khedhiri, Hadhami Ben Turkia, +8

Diagnostic Pathology

Phenotypic continuum of type 2 Gaucher's disease: an intermediate phenotype between perinatal-lethal and classic type 2 Gaucher's disease

Hadhami Ben Turkia, Néji Tebib, Hatem Azzouz, +4

Journal of Perinatology

Classic Type 2Intermediate PhenotypeGeneticsGenetic EpidemiologyType 2 +2