Ana Cristina Puga

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

Joseph Muenzer, Michael Beck, Christine M. Eng et al. · Genetics in Medicine · 2010 · 232 citations · Full text

Life Sciences, Biochemistry, Enzymology +1

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

Elena Lukina, Nora Watman, Elsa Ávila Arreguin et al. · Blood · 2010 · 157 citations · Full text

Medicine, Hematology, Pharmacotherapy +3

Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial

Timothy M. Cox, Guillermo Drelichman, Renata Cravo et al. · The Lancet · 2015 · 156 citations

Pharmacy, Internal Medicine, Pharmacotherapy +7

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

Elena Lukina, Nora Watman, Elsa Ávila Arreguin et al. · Blood · 2010 · 154 citations · Full text

Pharmacy, Pharmacotherapy, Oral Drug Delivery +3

Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months

Melissa Wasserstein, George A. Díaz, Robin Lachmann et al. · Journal of Inherited Metabolic Disease · 2018 · 121 citations · Full text

Medicine, Internal Medicine, Pharmacology +6