Publications by authors named "Patrick Yeramian"
Introduction: Sodium phenylbutyrate and taurursodiol (PB and TURSO) is hypothesized to mitigate endoplasmic reticulum stress and mitochondrial dysfunction, two of many mechanisms implicated in Alzheimer's disease (AD) pathophysiology.
Methods: The first-in-indication phase 2a PEGASUS trial was designed to gain insight into PB and TURSO effects on mechanistic targets of engagement and disease biology in AD. The primary clinical efficacy outcome was a global statistical test combining three endpoints relevant to disease trajectory (cognition [Mild/Moderate Alzheimer's Disease Composite Score], function [Functional Activities Questionnaire], and total hippocampal volume on magnetic resonance imaging).
Article Synopsis
- Coformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) has been shown to extend survival and slow decline in amyotrophic lateral sclerosis (ALS) patients.
- The CENTAUR trial aimed to assess whether PB/TURSO improved survival without the need for tracheostomy or ventilation and reduced the occurrence of first hospitalizations.
- Results indicated that patients treated with PB/TURSO had a 47% lower risk of key health events like death or needing ventilation compared to those on placebo, highlighting its potential benefits for ALS management.
An orally administered, fixed-dose coformulation of sodium phenylbutyrate-taurursodiol (PB-TURSO) significantly slowed functional decline in a randomized, placebo-controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long-term survival analysis of participants in CENTAUR. In CENTAUR, adults with ALS were randomized 2:1 to PB-TURSO or placebo.
View Article and Find Full Text PDFBackground: Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy and safety of a combination of the two compounds in persons with amyotrophic lateral sclerosis (ALS) are not known.
Methods: In this multicenter, randomized, double-blind trial, we enrolled participants with definite ALS who had had an onset of symptoms within the previous 18 months.
Article Synopsis
- The study found that adding trastuzumab to chemotherapy improves survival rates for patients with HER2(+) breast cancer by enhancing immune responses not just targeted at HER2, but also to other related proteins.
- Analysis of blood samples from 48 women revealed that many had elevated antibodies to specific cancer-related proteins before treatment, and after receiving trastuzumab and chemotherapy, a significant majority (69%) showed increased antibody responses, which correlated with better progression-free survival (PFS) and overall survival (OS).
- Interestingly, patients with high preexisting immunity to HER2-ICD did not benefit from the treatment, highlighting a complex relationship between the immune response and treatment efficacy.
Background: Sleeping sickness (human African trypanosomiasis [HAT]) is caused by protozoan parasites and characterized by a chronic progressive course, which may last up to several years before death. We conducted two Phase 2 studies to determine the efficacy and safety of oral pafuramidine in African patients with first stage HAT.
Methods: The Phase 2a study was an open-label, non-controlled, proof-of-concept study where 32 patients were treated with 100 mg of pafuramidine orally twice a day (BID) for 5 days at two trypanosomiasis reference centers (Angola and the Democratic Republic of the Congo [DRC]) between August 2001 and November 2004.
Background: DB289 is the orally active prodrug of the diamidine DB75, which was developed for the treatment of human African trypanosomiasis.
Methods: We tested the safety and efficacy of DB289 for the treatment of Plasmodium vivax and acute, uncomplicated P. falciparum infections in an open-label pilot study at the Hospital for Tropical Diseases in Bangkok.