Back to Authors

ONOFRJ, MARCO C.

0000-0002-0480-2495

A5041851539 | I39387349

Mayo Clinic

University of Chieti-Pescara

Citations 3

Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency

JAMA Network Open | September 24, 2021 | Volume: 4 | Issue: 9 | Pages: e2125584

10.1001/jamanetworkopen.2021.25584 | PMID 34559230

Prediction of on-treatment disability worsening in RRMS with the MAGNIMS score

Multiple Sclerosis Journal | July 8, 2020 | Volume: 27 | Issue: 5 | Pages: 695-705

10.1177/1352458520936823 | PMID 32639855

Background: The magnetic resonance imaging in multiple sclerosis (MAGNIMS) score combines relapses and magnetic resonance imaging (MRI) lesions to predict disability outcomes in relapsing–remitting multiple sclerosis (RRMS) treated with interferon-β. Objective: To validate the MAGNIMS score and extend to other disease-modifying therapies (DMTs). To examine the prognostic value of gadolinium contrast-enhancing (Gd+) lesions. Methods: This RRMS MSBase cohort study ( n = 2293) used a Cox model to examine the prognostic value of relapses, MRI activity and the MAGNIMS score for disability worsening during treatment with interferon-β and three other DMTs. Results: Three new T2 lesions (hazard ratio (HR) = 1.60, p = 0.028) or two relapses (HR = 2.24, p = 0.002) on interferon-β (for 12 months) were predictive of disability worsening over 4 years. MAGNIMS score = 2 (1 relapse and ⩾3 T2 lesions or ⩾2 relapses) was associated with a greater risk of disability worsening on interferon-β (HR = 2.0, p = 0.001). In pooled cohort of four DMTs, similar associations were seen (MAGNIMS score = 2: HR = 1.72, p = 0.001). Secondary analyses demonstrated that the addition of Gd+ to the MAGNIMS did not materially improve its prediction of disability worsening. Conclusion: We have validated the MAGNIMS score in RRMS and extended its application to three other DMTs: 1 relapse and ⩾3 T2 lesions or ⩾2 relapses predicted worsening of disability. Contrast-enhancing lesions did not substantially improve the prognostic score.
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

Alzheimer's & Dementia | October 18, 2019 | Volume: 15 | Issue: 7S_Part_23 | Pages: No page available

10.1016/j.jalz.2019.06.4746