Clinical outcome assessments of disease burden and progression in late-onset GM2 gangliosidoses

Authors: Kissell, Julie, Rochmann, Camille, Minini, Pascal, Eichler, Florian, Stephen, Christopher D., Lau, Heather, Toro, Camilo, Johnston, Jean M., Krupnick, Robert, Hamed, Alaa, Cox, Gerald F.

Source: In Molecular Genetics and Metabolism July 2024 142(3)

Safety and efficacy of venglustat in GBA1-associated Parkinson's disease: an international, multicentre, double-blind, randomised, placebo-controlled, phase 2 trial

Authors: Giladi, Nir, Alcalay, Roy N, Cutter, Gary, Gasser, Thomas, Gurevich, Tanya, Höglinger, Günter U, Marek, Kenneth, Pacchetti, Claudio, Schapira, Anthony H V, Scherzer, Clemens R, Simuni, Tanya, Minini, Pascal, Sardi, S Pablo, Peterschmitt, M Judith ^*

Source: In The Lancet Neurology August 2023 22(8):661-671

Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial

Authors: Gansevoort, Ronald T., Hariri, Ali, Minini, Pascal, Ahn, Curie, Chapman, Arlene B., Horie, Shigeo, Knebelmann, Bertrand, Mrug, Michal, Ong, Albert C.M., Pei, York P.C., Torres, Vicente E., Modur, Vijay, Antonshchuk, Igor, Perrone, Ronald D.

Source: In American Journal of Kidney Diseases May 2023 81(5):517-527

The STAGED-PKD 2-Stage Adaptive Study With a Patient Enrichment Strategy and Treatment Effect Modeling for Improved Study Design Efficiency in Patients With ADPKD

Authors: Perrone, Ronald D., Hariri, Ali, Minini, Pascal, Ahn, Curie, Chapman, Arlene B., Horie, Shigeo, Knebelmann, Bertrand, Mrug, Michal, Ong, Albert C.M., Pei, York P.C., Torres, Vicente E., Modur, Vijay, Gansevoort, Ronald T.

Source: In Kidney Medicine October 2022 4(10)

Lipoprotein(a) reductions from PCSK9 inhibition and major adverse cardiovascular events: Pooled analysis of alirocumab phase 3 trials

Authors: Ray, Kausik K., Vallejo-Vaz, Antonio J., Ginsberg, Henry N., Davidson, Michael H., Louie, Michael J., Bujas-Bobanovic, Maja, Minini, Pascal, Eckel, Robert H., Cannon, Christopher P.

Source: In Atherosclerosis September 2019 288:194-202

Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo in patients with heterozygous familial hypercholesterolemia

Authors: Kastelein, John J.P., Hovingh, G. Kees, Langslet, Gisle, Baccara-Dinet, Marie T., Gipe, Daniel A., Chaudhari, Umesh, Zhao, Jian, Minini, Pascal, Farnier, Michel

Source: In Journal of Clinical Lipidology January-February 2017 11(1):195-203

Effect of Alirocumab on Lipoprotein(a) Over ≥1.5 Years (from the Phase 3 ODYSSEY Program)

Authors: Gaudet, Daniel, Watts, Gerald F., Robinson, Jennifer G., Minini, Pascal, Sasiela, William J., Edelberg, Jay, Louie, Michael J., Raal, Frederick J.

Source: In The American Journal of Cardiology 1 January 2017 119(1):40-46

Efficacy of alirocumab in high cardiovascular risk populations with or without heterozygous familial hypercholesterolemia: Pooled analysis of eight ODYSSEY Phase 3 clinical program trials

Authors: Farnier, Michel, Gaudet, Daniel, Valcheva, Velichka, Minini, Pascal, Miller, Kathryn, Cariou, Bertrand

Source: In International Journal of Cardiology 15 November 2016 223:750-757

A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim AnalysisWhen Slope Is the Primary Endpoint.

Authors: Qi Zhang, Yuqian Shen, Hui Quan, Minini, Pascal, Lin Wang

Source: Statistics in Biopharmaceutical Research; Apr-Jun2024, Vol. 16 Issue 2, p218-228, 11p

AMETHIST: Baseline characteristics from a phase 3 trial of venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, in GM2-gangliosidoses and related diseases

Authors: Tifft, Cynthia J., Batsu, Isabela, Zheng, Riliang, Minini, Pascal

Source: In Molecular Genetics and Metabolism February 2023 138(2)

Changes in hematologic and visceral manifestations over time following imiglucerase initiation in Gaucher disease type 1 and type 3 pediatric patients in the ICGG Gaucher Registry

Authors: Mistry, Pramod K., Bianculli, Pablo, Batsu, Isabela, Heine, Walter, Oliveira-dos-Santos, Antonio, Minini, Pascal, Batista, Julie

Source: In Molecular Genetics and Metabolism February 2023 138(2)

Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for Gaucher disease type 3: Phase 3 LEAP2MONO trial design

Authors: Heine, Walter, Batsu, Isabela, Minini, Pascal, Cherkasov, Dmitry

Source: In Molecular Genetics and Metabolism February 2023 138(2)

Fine-motor functioning and its relevance to activities of daily living in adults with late-onset GM2 gangliosidosis: Analysis of baseline data of AMETHIST trial

Authors: Cox, Timothy M., Rodriguez, Maria B., Dyevre, Valérie, Msihid, Jerome, Gwaltney, Chad, Pulikottil-Jacob, Ruth, Zheng, Riliang, Minini, Pascal, Batsu, Isabela

Source: In Molecular Genetics and Metabolism February 2023 138(2)

Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3

Authors: Batsu, Isabela, Zheng, Riliang, Minini, Pascal, Cherkasov, Dmitry, Scott, David, Goker-Alpan, Ozlem, Hennermann, Julia B., Sakurai, Ken, Cox, Timothy M.

Source: In Molecular Genetics and Metabolism February 2023 138(2)

Bleeding Risk in Patients With Atrial Fibrillation: The AMADEUS Study

Authors: Lane, Deirdre A., Kamphuisen, Pieter W., Minini, Pascal, Büller, Harry R., Lip, Gregory Y.H.

Source: In Chest July 2011 140(1):146-155

Effects of a selective vasopressin V2 receptor antagonist, satavaptan, on ascites recurrence after paracentesis in patients with cirrhosis

Authors: Wong, Florence, Gines, Pere, Watson, Hugh, Horsmans, Yves, Angeli, Paolo, Gow, Paul, Minini, Pascal, Bernardi, Mauro

Source: In Journal of Hepatology August 2010 53(2):283-290

Venglustat in GBA1-related Parkinson's disease – Authors' reply

Authors: Giladi, Nir, Alcalay, Roy N, Cuer, Gary, Gasser, Thomas, Gurevich, Tanya, Höglinger, Günter U, Marek, Kenneth, PaccheE, Claudio, Schapira, Anthony H V, Scherzer, Clemens R, Simuni, Tanya, Minini, Pascal, Peterschmi, M Judith, Sardi, S Pablo

Source: In The Lancet Neurology February 2024 23(2):137-138

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

Authors: Schiffmann, Raphael¹, rschiffmann@texasneurology.com, Cox, Timothy M², Dedieu, Jean-François³, Gaemers, Sebastiaan J M⁴, Hennermann, Julia B⁵, Ida, Hiroyuki⁶, Mengel, Eugen^5,7, Minini, Pascal⁸, Mistry, Pramod⁹, Musholt, Petra B¹⁰, Scott, David¹¹, Sharma, Jyoti¹², Peterschmitt, M Judith¹³

Source: Brain: A Journal of Neurology; Feb2023, Vol. 146 Issue 2, p461-474, 14p

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, Placebo-Controlled MOVES-PD Trial.

Authors: 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

Source: Journal of Parkinson's Disease; 2022, Vol. 12 Issue 2, p557-570, 14p

Subject Terms: PARKINSON'S disease, PHARMACODYNAMICS, PHARMACOKINETICS, GENETIC mutation, PANIC attacks

Pharmacokinetics and biomarker responses in patients with Gaucher disease type 3 or GBA-associated Parkinson's disease treated with venglustat

Authors: Peterschmitt, M. Judith, Sharma, Jyoti, Karetsky, Lina, Ji, Allena, Pandya, Shirali, Freisens, Selena, Minini, Pascal

Source: In Molecular Genetics and Metabolism February 2022 135(2):S96-S96