Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1

por Peterschmitt, M. Judith, Freisens, Selena, Underhill, Lisa H., Foster, Meredith C., Lewis, Grace, Gaemers, Sebastiaan J. M.
Publicado 2019

Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers

por Peterschmitt, M. Judith, Crawford, Nigel P. S., Gaemers, Sebastiaan J. M., Ji, Allena J., Sharma, Jyoti, Pham, Theresa T.
Publicado 2020

Pregnancy outcome in women with Gaucher disease type 1 who had unplanned pregnancies during eliglustat clinical trials

por Lukina, Elena, Balwani, Manisha, Belmatoug, Nadia, Watman, Nora, Hughes, Derralynn, Gaemers, Sebastiaan J. M., Foster, Meredith C., Lewis, Grace, Peterschmitt, M. Judith
Publicado 2020

Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial

por Lukina, Elena, Watman, Nora, Dragosky, Marta, Lau, Heather, Avila Arreguin, Elsa, Rosenbaum, Hanna, Zimran, Ari, Foster, Meredith C., Gaemers, Sebastiaan J. M., Peterschmitt, M. Judith
Publicado 2018

Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial

por Pleat, Rebecca, Cox, Timothy M., Burrow, T. Andrew, Giraldo, Pilar, Goker-Alpan, Ozlem, Rosenbloom, Barry E., Croal, Laura R., Underhill, Lisa H., Gaemers, Sebastiaan J.M., Peterschmitt, M. Judith
Publicado 2016

Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial

por Schiffmann, Raphael, Cox, Timothy M, Dedieu, Jean-François, Gaemers, Sebastiaan J M, Hennermann, Julia B, Ida, Hiroyuki, Mengel, Eugen, Minini, Pascal, Mistry, Pramod, Musholt, Petra B, Scott, David, Sharma, Jyoti, Peterschmitt, M Judith
Publicado 2022

Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial

por Mistry, Pramod K., Lukina, Elena, Ben Turkia, Hadhami, Shankar, Suma P., Baris, Hagit, Ghosn, Marwan, Mehta, Atul, Packman, Seymour, Pastores, Gregory, Petakov, Milan, Assouline, Sarit, Balwani, Manisha, Danda, Sumita, Hadjiev, Evgueniy, Ortega, Andres, Gaemers, Sebastiaan J. M., Tayag, Regina, Peterschmitt, M. Judith
Publicado 2017

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

por Mistry, Pramod K., Lukina, Elena, Ben Turkia, Hadhami, Shankar, Suma P., Baris Feldman, Hagit, Ghosn, Marwan, Mehta, Atul, Packman, Seymour, Lau, Heather, Petakov, Milan, Assouline, Sarit, Balwani, Manisha, Danda, Sumita, Hadjiev, Evgueniy, Ortega, Andres, Foster, Meredith C., Gaemers, Sebastiaan J. M., Peterschmitt, M. Judith
Publicado 2021

Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson’s Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Pl...

por Peterschmitt, M. Judith, Saiki, Hidemoto, Hatano, Taku, Gasser, Thomas, Isaacson, Stuart H., Gaemers, Sebastiaan J.M., Minini, Pascal, Saubadu, Stéphane, Sharma, Jyoti, Walbillic, Samantha, Alcalay, Roy N., Cutter, Gary, Hattori, Nobutaka, Höglinger, Günter U., Marek, Kenneth, Schapira, Anthony H.V., Scherzer, Clemens R., Simuni, Tanya, Giladi, Nir, Sardi, Sergio Pablo, Fischer, Tanya Z.
Publicado 2022