CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.

Importance: Bioenergetic failure has been proposed as a driver of amyotrophic lateral sclerosis (ALS). CNM-Au8 is a suspension of gold nanocrystals that catalyzes the conversion of nicotinamide adenine dinucleotide hydride into NAD+, resulting in an increase of cellular adenosine triphosphate production.

Objective: To determine the effects of CNM-Au8 on ALS disease progression.

Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.

Importance: Amyotrophic lateral sclerosis (ALS) is a fatal disease. The sigma-1 (σ1) receptor emerged as a target for intervention.

Objective: To determine the effects of pridopidine, a σ1-receptor agonist, in ALS.

Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial.

Importance: Myeloperoxidase is one of the most abundant peroxidase enzymes in activated myeloid cells. Myeloperoxidase inhibitors may have a clinical benefit in amyotrophic lateral sclerosis (ALS) by slowing neurodegeneration via reduced neuroinflammation and oxidative stress.

Objective: To determine the safety, tolerability, and efficacy of verdiperstat, a selective myeloperoxidase inhibitor, in ALS.

Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.

Importance: The etiology of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, is unknown. However, neuroinflammation and complement activation may play a role in disease progression.

Objective: To determine the effects of zilucoplan, an inhibitor of complement C5, in individuals with ALS.

Learning to Train and to Explain a Deep Survival Model with Large-Scale Ovarian Cancer Transcriptomic Data.

Ovarian cancer is a complex disease with poor outcomes that affects women worldwide. The lack of successful therapeutic options for this malignancy has led to the need to identify novel biomarkers for patient stratification. Here, we aim to develop the outcome predictors based on the gene expression data as they may serve to identify categories of patients who are more likely to respond to certain therapies.