Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collabor...

por Ibrahim, Jennifer, Underhill, Lisa H., Taylor, John S., Angell, Jennifer, Peterschmitt, M. Judith
Publicado 2016

Effects of paroxetine, ketoconazole, and rifampin on the metabolism of eliglustat, an oral substrate reduction therapy for Gaucher disease type 1

por Vu, Lucie, Cox, Gerald F., Ibrahim, Jennifer, Peterschmitt, M. Judith, Ross, Leorah, Thibault, Nathan, Turpault, Sandrine
Publicado 2020

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

Prospective study of the natural history of chronic acid sphingomyelinase deficiency in children and adults: eleven years of observation

por McGovern, Margaret M., Wasserstein, Melissa P., Bembi, Bruno, Giugliani, Roberto, Mengel, K. Eugen, Vanier, Marie T., Zhang, Qi, Peterschmitt, M. Judith
Publicado 2021

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

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat

por Kamath, Ravi S., Lukina, Elena, Watman, Nora, Dragosky, Marta, Pastores, Gregory M., Arreguin, Elsa Avila, Rosenbaum, Hanna, Zimran, Ari, Aguzzi, Rasha, Puga, Ana Cristina, Norfleet, Andrea M., Peterschmitt, M. Judith, Rosenthal, Daniel I.
Publicado 2014

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

A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat

por Abrams, Ruth, Kaddi, Chanchala D., Tao, Mengdi, Leiser, Randolph J., Simoni, Giulia, Reali, Federico, Tolsma, John, Jasper, Paul, van Rijn, Zachary, Li, Jing, Niesner, Bradley, Barrett, Jeffrey S., Marchetti, Luca, Peterschmitt, M. Judith, Azer, Karim, Neves‐Zaph, Susana
Publicado 2020

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