Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial

Autor: Peter A. Ljubenkov, Mary Koestler, Florence Pasquier, Dana Hilt, Beth Ann Haynes, Renaud La Joie, Lauren Edwards, Bradley F. Boeve, Jacob Hesterman, Ira J. Goodman, Leonardo Iaccarino, Rik Vandenberghe, Catherine J. Mummery, Marco Onofrj, Sophie Auriacombe, Henry Riordan, Gerhard Koenig, Julio C. Rojas, Hans J Moebius, Adam L. Boxer, Giovanni B. Frisoni, Scott M. McGinnis, Gary Wisniewski, Ken Marek, Didier Hannequin, Murray Grossman, John C. van Swieten, Holger Patzke, Isabelle Le Ber, Baruch Harris, Barbara Borroni

Publikováno v: JAMA Network Open
JAMA Network Open, American Medical Association, 2021, 4 (9), pp.e2125584. ⟨10.1001/jamanetworkopen.2021.25584⟩
JAMA Network Open, 2021, 4 (9), pp.e2125584. ⟨10.1001/jamanetworkopen.2021.25584⟩
JAMA network open, 4(9):25584. American Medical Association

Key Points Question What is the safety, tolerability, pharmacodynamic, and pharmacokinetic profile of the histone deacetylase inhibitor FRM-0334 in participants with progranulin gene (GRN) haploinsufficiency? Findings In this randomized placebo-contr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::85461c1fe333ce116f09c9a714feab20
http://hdl.handle.net/20.500.12278/123771

Safety and Tolerability of Multiple Ascending Doses of PRX002/RG7935, an Anti-α-Synuclein Monoclonal Antibody, in Patients With Parkinson Disease: A Randomized Clinical Trial

Autor: Frank G. Boess, Susanne Ostrowitzki, Beth Safirstein, Joseph Jankovic, Meret Martin-Facklam, Omid Omidvar, Jay Soto, Stuart Isaacson, Tonya K. Marmon, Aaron Ellenbogen, Sue G. Griffith, Joseph F. Quinn, Michael Grundman, Dale Schenk, Daniel Ness, Gene G. Kinney, Wagner Zago, Ira J. Goodman, Martin Koller

Publikováno v: JAMA neurology. 75(10)

Importance Aggregated α-synuclein is believed to be central to the pathogenesis of Parkinson disease (PD). PRX002/RG7935 (PRX002) is a humanized monoclonal antibody designed to target aggregated forms of α-synuclein, thereby inhibiting neuron-to-ne

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d1d5b85efcf3beacd5fa15868dc4d355
https://pubmed.ncbi.nlm.nih.gov/29913004

O4-02-01: PHASE 2A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF THE HISTONE DEACETYLASE INHIBITOR (HDACI), FRM-0334, IN ASYMPTOMATIC CARRIERS OF, OR PATIENTS WITH FRONTOTEMPORAL LOBAR DEGENERATION (FTLD) DUE TO, PROGRANULIN GENE MUTATIONS

Autor: Marco Onofrj, Holger Patzke, Murray Grossman, Rik Vandenberghe, Henry Riordan, Isabelle Le Ber, Sophie Auriacombe, Jacob Hesterman, Hans J Moebius, Gerhard Koenig, Giovanni B. Frisoni, Adam L. Boxer, Bradley F. Boeve, Gary Wisniewski, Ken Marek, Ira J. Goodman, Florence Pasquier, Didier Hannequin, Baruch Harris, Barbara Borroni, Scott M. McGinnis, Dana Hilt, John C. van Swieten, Catherine J. Mummery

Publikováno v: Alzheimer's & Dementia. 15:P1231-P1232

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::0b37d602fc3682ef9049e843e9680266
https://doi.org/10.1016/j.jalz.2019.06.4746

[P4–487]: PATIENT ENGAGEMENT IN ALZHEIMER's AND MILD COGNITIVE IMPAIRMENT CLINICAL TRIALS

Autor: Craig Curtis, Ira J. Goodman, Marieke I. Cajal

Publikováno v: Alzheimer's & Dementia. 13

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::004ba025ca46843242ab6a92345f424e
https://doi.org/10.1016/j.jalz.2017.07.648

P4-663: INITIATION OF THE PHASE 2/3 GAIN TRIAL OF COR388, A NOVEL BACTERIAL VIRULENCE FACTOR INHIBITOR FOR THE TREATMENT OF ALZHEIMER'S DISEASE (AD) BASED ON PHASE I A/B SAFETY, PK, BIOMARKER AND EFFICACY DATA

Autor: Ira J. Goodman, Mark I. Ryder, Stephen G. Thein, Ursula Haditsch, Mike Detke, Dave Hennings, Leslie J. Holsinger, Shirin Kapur, Steve Dominy, Casey C. Lynch, Florian Ermini, Debasish Raha

Publikováno v: Alzheimer's & Dementia. 15:P1585-P1585

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::80466dde04acca93c34519ea527d9783
https://doi.org/10.1016/j.jalz.2019.09.029