HIMALAYA: learning from another negative phase 2 trial in ALS

Recent advances in the diagnosis and prognosis of amyotrophic lateral sclerosis

10.1016/s1474-4422(21)00465-8 · 2022

Emerging insights into the complex genetics and pathophysiology of amyotrophic lateral sclerosis

10.1016/s1474-4422(21)00414-2 · 2022

TDP-43: [GU]-ardian of the transcriptome

10.1186/s13024-026-00944-2 · 2026

Rodent models of amyotrophic lateral sclerosis

10.1002/0471141755.ph0567s69 · 2015

Single-cell transcriptomic and functional studies identify glial state changes and a role for inflammatory RIPK1 signaling in ALS pathogenesis

10.1016/j.immuni.2025.02.024 · 2025

RIPK1 mediates axonal degeneration by promoting inflammation and necroptosis in ALS

10.1126/science.aaf6803 · 2016

Safety, pharmacokinetics, and target engagement of a brain penetrant RIPK1 inhibitor, SAR443820 (DNL788), in healthy adult participants

10.1111/cts.13690 · 2024

Safety, tolerability, and efficacy of RIPK1 inhibitor, SAR443820, in amyotrophic lateral sclerosis (HIMALAYA): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

10.1016/s1474-4422(26)00277-2 · 2026

Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial